1. BOSTON, Sept. 22, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases of abnormal mineralization, today announced Axel Bolte, MSc, MBA, the company's co-founder, president, and chief executive officer, will present at the 2021 Cantor Virtual Global Healthcare Conference on Wednesday, September 29, at 1:20 p.m. ET.

    The presentation will be webcast live and can be accessed from the Investors & News section of Inozyme's website under Events. An archived replay of the webcast will be available for up to 60 days following the event.

    About Inozyme Pharma
    Inozyme Pharma, Inc. (NASDAQ:INZY), is a rare disease biopharmaceutical company…

    BOSTON, Sept. 22, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases of abnormal mineralization, today announced Axel Bolte, MSc, MBA, the company's co-founder, president, and chief executive officer, will present at the 2021 Cantor Virtual Global Healthcare Conference on Wednesday, September 29, at 1:20 p.m. ET.

    The presentation will be webcast live and can be accessed from the Investors & News section of Inozyme's website under Events. An archived replay of the webcast will be available for up to 60 days following the event.

    About Inozyme Pharma

    Inozyme Pharma, Inc. (NASDAQ:INZY), is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 Deficiencies.

    Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

    Contacts

    Investors:

    Inozyme Pharma

    Stefan Riley, Director of Investor Relations

    stefan.riley@inozyme.com    

    Media:

    SmithSolve

    Alex Van Rees

    (973) 442-1555 ext. 111

    alex.vanrees@smithsolve.com



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  2. - Received Orphan Drug Designation from the European Medicines Agency for INZ-701 for the treatment of ABCC6 Deficiency –

    – Clinical Trial Application in Europe accepted for Phase 1/2 trial of INZ-701 in ABCC6 Deficiency –

    – Investigational New Drug application in U.S. accepted for Phase 1/2 trial of INZ-701 in ABCC6 Deficiency –

    - Expect to enroll patients in Phase 1/2 clinical trials in ENPP1 Deficiency and ABCC6 Deficiency in Q4 2021 and report preliminary biomarker and safety data in the first half of 2022 –

    - Cash, cash equivalents, and investments expected to support continued operations into the fourth quarter of 2022 –

    BOSTON, Aug. 11, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical…

    - Received Orphan Drug Designation from the European Medicines Agency for INZ-701 for the treatment of ABCC6 Deficiency –

    – Clinical Trial Application in Europe accepted for Phase 1/2 trial of INZ-701 in ABCC6 Deficiency –

    – Investigational New Drug application in U.S. accepted for Phase 1/2 trial of INZ-701 in ABCC6 Deficiency –

    - Expect to enroll patients in Phase 1/2 clinical trials in ENPP1 Deficiency and ABCC6 Deficiency in Q4 2021 and report preliminary biomarker and safety data in the first half of 2022 –

    - Cash, cash equivalents, and investments expected to support continued operations into the fourth quarter of 2022 –

    BOSTON, Aug. 11, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today reported financial results for the second quarter ended June 30, 2021, and provided recent business highlights.

    "We achieved several important milestones during the second quarter of 2021. We expect to initiate our clinical trials in ENPP1 Deficiency and ABCC6 Deficiency in the fourth quarter of this year and then to report preliminary biomarker and safety data in the first half of 2022," said Axel Bolte, MSc, MBA, Inozyme's co-founder, president, and chief executive officer. "We continue to make progress towards our mission of bringing hope to patients and their families who are living with ENPP1 Deficiency and ABCC6 Deficiency. In addition, we added several talented members to our leadership team who will contribute to this next phase of our growth."

    Recent Business Highlights

    • Clinical Trial Application (CTA) in Europe and Investigational New Drug (IND) application in U.S. for ABCC6 Deficiency accepted – The Company expects to enroll patients in a Phase 1/2 clinical trial in the fourth quarter of 2021 and report preliminary biomarker and safety data in the first half of 2022.
    • Published data from Natural History Study of ENPP1 and ABCC6 Deficiencies – Peer-reviewed article in the Journal of Bone and Mineral Research shows early mortality risk in GACI patients despite attempts to treat with bisphosphonates, high prevalence of rickets almost exclusive to ENPP1 Deficiency, and a spectrum of heterogenous calcification and multiple organ complications with both ENPP1 and ABCC6 variants, which suggests an overlapping pathology.
    • Appointed Gayle Gironda as senior vice president, human resources – Ms. Gironda is a human resources leader with more than 20 years of experience in organizational design, talent recruitment, performance culture, planning and leadership development.
    • Appointed David Thompson, M.A., M.S., Ph.D. as chief development officer – Dr. Thompson has more than 30 years of experience designing and leading research and development programs focused on bone disorders and phosphate regulation. He previously served as Inozyme's senior vice president and chief scientific officer from 2018 to 2020 and was responsible for scientific research as the Company built its proprietary pipeline of investigational therapies, beginning with INZ-701.

    Upcoming Anticipated Milestones

    The Company also announced the following anticipated milestones for the INZ-701 clinical development program, subject to COVID-19-related restrictions:

    • ENPP1 Deficiency
      • Q4 2021: Start enrollment for Phase 1/2 clinical trial
      • Q1 2022: Initiate prospective natural history study
      • H1 2022: Report preliminary safety and biomarker data from Phase 1/2 clinical trial
    • ABCC6 Deficiency
      • Q4 2021: Start enrollment for Phase 1/2 clinical trial
      • H1 2022: Report preliminary safety and biomarker data from Phase 1/2 clinical trial

    Financial Results for the Quarter Ended Jun 30, 2021

    • Cash Position and Financial Guidance – Cash, cash equivalents, and investments were $137.5 million as of June 30, 2021. Based on its current plans, the Company expects that its existing cash, cash equivalents, and investments will be sufficient to enable funding of its operating expenses and capital expenditure requirements into the fourth quarter of 2022.
    • Research and Development (R&D) Expenses – R&D expenses were $8.2 million for the quarter ended June 30, 2021, compared to $7.9 million for the quarter ended June 30, 2020. The increase was primarily due to increased salaries, employee-related costs, and stock-based compensation expense due to the growth in the number of R&D employees. These increases were partially offset by a decrease in preclinical toxicology studies in support of our IND filing for INZ-701, and lower manufacturing costs based on the timing of production runs.
    • General and Administrative (G&A) Expenses – G&A expenses were $4.4 million for the quarter ended June 30, 2021, compared to $1.7 million for the quarter ended June 30, 2020. The increase was primarily due to the growth in the number of G&A employees, an increase in legal fees related to new contracts and operations as a public company and generally higher fees in areas such as audit, tax, and information technology to support the Company's growth.
    • Net Loss – Net loss was $12.5 million, or $0.53 loss per share, for the quarter ended June 30, 2021, compared to $9.5 million, or $7.57 loss per share, for the quarter ended June 30, 2020.

    About Inozyme Pharma

    Inozyme Pharma, Inc. (NASDAQ:INZY), is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 Deficiencies.

    Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation and timing of our clinical trials, the initiation and timing of our natural history study, our research and development programs, the availability of preclinical study and clinical trial data, the timing of our regulatory applications and the period over which we believe that our existing cash, cash equivalents and investments will be sufficient to fund our operating expenses. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to initiate its planned Phase 1/2 clinical trials of INZ-701 for ENPP1 Deficiency and ABCC6 Deficiency; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section in the Company's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Condensed Consolidated Balance Sheet Data

    (Unaudited)

    (in thousands)

     June 30,

    2021
     December 31,

    2020
    Cash, cash equivalents and investments$137,464  $159,896 
    Total assets 148,259   169,363 
    Total liabilities 9,985   11,260 
    Additional paid-in-capital 252,920   249,175 
    Accumulated deficit (114,666)  (91,076)
    Total stockholders' equity 138,274   158,103 
            

    Condensed Consolidated Statements of Operations and Comprehensive Loss

    (Unaudited)

    (in thousands, except share and per share data)

     Three Months Ended June 30,
      2021   2020 
    Operating expenses:   
    Research and development$8,220  $7,877 
    General and administrative 4,435   1,671 
    Total operating expenses 12,655   9,548 
    Loss from operations (12,655)  (9,548)
    Other income (expense):   
    Interest income 58   71 
    Other income (expense) 57   4 
    Other income (expense), net 115   75 
    Net loss$(12,540) $(9,473)
    Other comprehensive income (loss):   
    Unrealized gains (losses) on available-for-sale securities 6   (15)
    Total other comprehensive income (loss) 6   (15)
    Comprehensive loss$(12,534) $(9,488)
    Net loss attributable to common stockholders—basic and diluted$(12,540) $(9,473)
    Net loss per share attributable to common stockholders—basic and diluted$(0.53) $(7.57)
    Weighted-average common shares outstanding—basic and diluted 23,490,591   1,251,244 



     Six Months Ended June 30,
      2021   2020 
    Operating expenses:   
    Research and development$14,823  $14,283 
    General and administrative 8,804   3,171 
    Total operating expenses 23,627   17,454 
    Loss from operations (23,627)  (17,454)
    Other income (expense):   
    Interest income 121   242 
    Other income (expense) (84)  1 
    Other income (expense), net 37   243 
    Net loss$(23,590) $(17,211)
    Other comprehensive income (loss):   
    Unrealized gains (losses) on available-for-sale securities 16   8 
    Total other comprehensive income (loss) 16   8 
    Comprehensive loss$(23,574) $(17,203)
    Net loss attributable to common stockholders—basic and diluted$(23,590) $(17,211)
    Net loss per share attributable to common stockholders—basic and diluted$(1.01) $(14.01)
    Weighted-average common shares outstanding—basic and diluted 23,460,218   1,228,296 
        

    Contacts

    Investors:

    Inozyme Pharma

    Stefan Riley, Director of Investor Relations

    Stefan.riley@inozyme.com

    Media:

    Alex Van Rees, SmithSolve

    (973) 442-1555 ext. 111

    Alex.vanrees@smithsolve.com



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  3. BOSTON, Aug. 04, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced Axel Bolte, MSc, MBA, the company's co-founder, president, and chief executive officer, will participate in a panel at the 2021 Wedbush PacGrow Healthcare Virtual Conference on Wednesday, Aug. 11. Members of the Inozyme management team will also host investor meetings during the conference.

    2021 Wedbush PacGrow Healthcare Virtual Conference

    • Panel: UltraOrphan – When You're One in a Million
    • Date: Wednesday, Aug. 11
    • Time: 2:55-3:25 p.m. ET

    About Inozyme Pharma
    Inozyme Pharma (NASDAQ:INZY) is a rare disease biopharmaceutical company developing…

    BOSTON, Aug. 04, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced Axel Bolte, MSc, MBA, the company's co-founder, president, and chief executive officer, will participate in a panel at the 2021 Wedbush PacGrow Healthcare Virtual Conference on Wednesday, Aug. 11. Members of the Inozyme management team will also host investor meetings during the conference.

    2021 Wedbush PacGrow Healthcare Virtual Conference

    • Panel: UltraOrphan – When You're One in a Million
    • Date: Wednesday, Aug. 11
    • Time: 2:55-3:25 p.m. ET

    About Inozyme Pharma

    Inozyme Pharma (NASDAQ:INZY) is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 Deficiencies.

    Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

    Contacts

    Investors:

    Inozyme Pharma

    Stefan Riley, Director of Investor Relations

    Stefan.riley@inozyme.com

    Media:

    SmithSolve

    Alex Van Rees

    (973) 442-1555 ext. 111

    Alex.vanrees@smithsolve.com

     



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  4. BOSTON, July 21, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation to INZ-701 for the treatment of ABCC6 Deficiency. INZ-701, an investigational enzyme replacement therapy (ERT), was granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) and the EMA for the treatment of ENPP1 Deficiency.

    ABCC6 Deficiency is a rare, inherited disorder that can present as generalized arterial calcification of infancy (GACI) type 2 in infants and as pseudoxanthoma elasticum (PXE) in children and adults. It is one of…

    BOSTON, July 21, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation to INZ-701 for the treatment of ABCC6 Deficiency. INZ-701, an investigational enzyme replacement therapy (ERT), was granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) and the EMA for the treatment of ENPP1 Deficiency.

    ABCC6 Deficiency is a rare, inherited disorder that can present as generalized arterial calcification of infancy (GACI) type 2 in infants and as pseudoxanthoma elasticum (PXE) in children and adults. It is one of several disorders that features a significant decrease in plasma pyrophosphate (PPi) levels, a potent regulator of mineralization. In patients with ABCC6 Deficiency, the abnormal calcification caused by low PPi can result in vision loss and life-threatening cardiovascular complications, among other morbidities. There is no approved treatment for ABCC6 Deficiency.

    "Obtaining Orphan Drug Designation from the EMA for the treatment of ABCC6 Deficiency marks another important step toward providing relief for patients who currently have no treatment options and positions INZ-701 to become the first-ever available therapy for this condition," said Axel Bolte, MSc, MA, co-founder, president, and chief executive officer of Inozyme.

    Orphan Drug Designation in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products (COMP). To qualify, a therapeutic candidate must be intended to treat a serious condition that affects fewer than five in 10,000 people in the EU, and there must be sufficient data to suggest the candidate may produce clinically relevant outcomes. The designation could allow for drug development incentives, including clinical protocol assistance, access to the centralized authorization procedure, reduced regulatory fees, and a ten-year period of market exclusivity in the EU after product approval.

    About INZ-701

    INZ-701 is an ENPP1 enzyme replacement therapy (ERT) in development for the treatment of mineralization disorders of the circulatory system, bones, and kidneys. In preclinical studies, the experimental therapy has shown potential to generate plasma pyrophosphate (PPi) and to restore it to appropriate physiological levels, thereby preventing calcification in the vasculature and kidneys, while at the same time correcting bone abnormalities. Inozyme is developing INZ-701 for certain rare, life-threatening, and devastating genetic disorders such as ENPP1 Deficiency and ABCC6 Deficiency in which PPi levels are below the normal physiological levels.

    Inozyme is preparing to initiate a Phase 1/2 clinical trial in patients with ENPP1 Deficiency and a separate Phase 1/2 clinical trial in patients with ABCC6 Deficiency in mid-2021.

    About ABCC6 Deficiency

    ABCC6 Deficiency is a rare, severe, inherited disorder caused by mutations in the ABCC6 gene, leading to low levels of PPi. PPi is essential for preventing harmful soft tissue calcification and regulating bone mineralization. ABCC6 Deficiency is a systemic and progressively debilitating condition estimated to affect more than 67,000 individuals worldwide. The condition is characterized by pathological mineralization in blood vessels and soft tissues throughout the body that can drive devastating medical problems.

    Some infants with ABCC6 Deficiency are diagnosed with generalized arterial calcification of infancy (GACI) type 2, a vascular condition that resembles GACI type 1, the acute infantile form of ENPP1 Deficiency. In older patients, ABCC6 Deficiency presents as pseudoxanthoma elasticum (PXE), a rare, inherited disorder in which individuals develop calcification of soft connective tissues, including in the eyes, cardiovascular system, and skin. There is no approved treatment for ABCC6 Deficiency.

    About Inozyme Pharma

    Inozyme Pharma (NASDAQ:INZY) is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 Deficiencies.

    Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the potential of our lead product candidate, INZ-701, the initiation and timing of our future clinical trials and our research and development programs. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to initiate its planned Phase 1/2 clinical trials of INZ-701 for ENPP1 Deficiency and ABCC6 Deficiency; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties, and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contacts

    Investors:

    Inozyme Pharma

    Stefan Riley, Director of Investor Relations

    Stefan.riley@inozyme.com

    Media:

    SmithSolve

    Alex Van Rees

    (973) 442-1555 ext. 111

    Alex.vanrees@smithsolve.com



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  5. BOSTON, July 13, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced that Gayle Gironda has been appointed as senior vice president of human resources. Ms. Gironda is a human resources leader with more than 20 years of experience in organizational design, talent recruitment, performance culture, planning and leadership development.

    "Gayle brings a deep knowledge of life sciences, stemming from her work across large multinational pharmaceutical organizations and small biotech companies, and a passion for supporting people living with rare diseases," said Axel Bolte, MSc, MBA, co-founder, president and…

    BOSTON, July 13, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced that Gayle Gironda has been appointed as senior vice president of human resources. Ms. Gironda is a human resources leader with more than 20 years of experience in organizational design, talent recruitment, performance culture, planning and leadership development.

    "Gayle brings a deep knowledge of life sciences, stemming from her work across large multinational pharmaceutical organizations and small biotech companies, and a passion for supporting people living with rare diseases," said Axel Bolte, MSc, MBA, co-founder, president and chief executive officer of Inozyme Pharma. "We are thrilled to welcome her to the team as we prepare to transition to a clinical-stage company with a commensurate expansion of our operational capabilities and growing talent pool. Gayle's experience will be essential as we position Inozyme for a new working environment while continuing to build our culture, which is focused on our patient communities and guides our mission of delivering first-in-class treatments for rare diseases without approved medicines."

    Most recently, Ms. Gironda served as vice president, human resources, global hematology and global market access at Bristol Myers Squibb (BMS), playing a key role in integrating the Hematology Business Unit as part of the company's acquisition of Celgene. Additionally, she supported the Global Market Access, Pricing and Health Economics and Outcomes Research (HEOR) teams through post-acquisition strategy, organizational design and talent alignment.

    Before joining Celgene in 2018 as vice president, human resources, global franchises, Ms. Gironda was executive director, HR, commercial operations at Alexion Pharmaceuticals. Her previous experience also includes leadership roles in human resources and operations for small-to-mid-sized companies such as Watson/Actavis, Jerini Ophthalmic, Inc. and Eyetech Pharmaceuticals.

    "Inozyme's leadership is committed to a collaborative and inclusive culture in which the entire team is deeply connected to the goal of treating patients with rare diseases," Ms. Gironda said. "I'm proud to join the company during such a critical time and look forward to playing a role in its continued growth."

    About Inozyme Pharma

    Inozyme Pharma (NASDAQ:INZY) is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 Deficiencies.

    Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the potential of our lead product candidate, INZ-701, the initiation and timing of our future clinical trials and our research and development programs. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to initiate its planned Phase 1/2 clinical trials of INZ-701 for ENPP1 Deficiency and ABCC6 Deficiency; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties, and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contacts

    Investors:

    Inozyme Pharma

    Stefan Riley, Director of Investor Relations

    Stefan.riley@inozyme.com

    Media:

    SmithSolve

    Alex Van Rees

    (973) 442-1555 ext. 111

    Alex.vanrees@smithsolve.com



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  6. - Clinical trial initiation expected in mid-2021 –
    - Preliminary safety and biomarker data expected by the end of 2021 -

    BOSTON, June 09, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced the acceptance of its Clinical Trial Application (CTA) from the National Agency for the Safety of Medicines and Health Products (ANSM) in France to allow initiation of its Phase 1/2 clinical trial of INZ-701, as a potential treatment for ABCC6 Deficiency. Inozyme plans to initiate its Phase 1/2 trial in mid-2021. This CTA was submitted and accepted as part of ANSM's Fast Track procedure designed to reduce processing…

    - Clinical trial initiation expected in mid-2021 –

    - Preliminary safety and biomarker data expected by the end of 2021 -

    BOSTON, June 09, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced the acceptance of its Clinical Trial Application (CTA) from the National Agency for the Safety of Medicines and Health Products (ANSM) in France to allow initiation of its Phase 1/2 clinical trial of INZ-701, as a potential treatment for ABCC6 Deficiency. Inozyme plans to initiate its Phase 1/2 trial in mid-2021. This CTA was submitted and accepted as part of ANSM's Fast Track procedure designed to reduce processing times for clinical trial authorization requests for innovative medical products.

    ABCC6 Deficiency is a rare, inherited disorder that can present as generalized arterial calcification of infancy (GACI) type 2 in infants and as pseudoxanthoma elasticum (PXE) in children and adults. This is one of several disorders with a significant decrease in plasma pyrophosphate (PPi) levels, a potent regulator of mineralization. In patients with ABCC6 Deficiency, the abnormal calcification caused by low PPi can result in vision loss and life-threatening cardiovascular complications, among other morbidities. There is no approved treatment for ABCC6 Deficiency.

    "The acceptance of this CTA marks another important regulatory milestone for Inozyme and a key advancement for INZ-701 as a potential treatment for people living with ABCC6 Deficiency," said Axel Bolte, MSc, MBA, co-founder, president, and chief executive officer of Inozyme Pharma. "We are well-positioned to execute on our planned clinical study and the notable level of interest from the ABCC6 Deficiency community underscores the urgent need for therapeutic options. I want to express my thanks to the team at Inozyme and our external collaborators, all of whom have been instrumental in our continued progress."

    INZ-701 is an ENPP1 enzyme replacement therapy (ERT) in development for the treatment of mineralization disorders of the circulatory system, bones, and kidneys. In preclinical studies, the experimental therapy has shown potential to generate PPi and to restore it to appropriate physiological levels, thereby preventing calcification in the vasculature and kidneys, while at the same time correcting bone abnormalities in Enpp1-deficient mice. In Abcc6-deficient mice, subcutaneous administration of INZ-701 (2 and 10 mg/kg every other day for two or eight weeks) led to a dose-dependent increase in plasma PPi levels at both two and eight weeks after initiation of treatment, leading to significantly lower levels of soft tissue mineralization.

    About ABCC6 Deficiency

    ABCC6 Deficiency is a rare, severe, inherited disorder caused by mutations in the ABCC6 gene, leading to low levels of PPi. PPi is essential for preventing harmful soft tissue calcification and regulating bone mineralization. ABCC6 Deficiency is a systemic and progressively debilitating condition estimated to affect more than 67,000 individuals worldwide. The condition is characterized by pathological mineralization in blood vessels and soft tissues throughout the body that can drive devastating medical problems.

    Some infants with ABCC6 Deficiency are diagnosed with generalized arterial calcification of infancy (GACI) type 2, a vascular condition that resembles GACI type 1, the acute infantile form of ENPP1 Deficiency. In older patients, ABCC6 Deficiency presents as pseudoxanthoma elasticum (PXE), a rare, inherited disorder in which individuals develop calcification of soft connective tissues, including in the eyes, cardiovascular system, and skin. There is no approved treatment for ABCC6 Deficiency.

    About INZ-701

    INZ-701 is an ENPP1 enzyme replacement therapy (ERT) in development for the treatment of mineralization disorders of the circulatory system, bones, and kidneys. In preclinical studies, the experimental therapy has shown potential to generate plasma pyrophosphate (PPi) and to restore it to appropriate physiological levels, thereby preventing calcification in the vasculature and kidneys, while at the same time correcting bone abnormalities. Inozyme is developing INZ-701 for certain rare, life-threatening, and devastating genetic disorders such as ENPP1 Deficiency and ABCC6 Deficiency in which PPi levels are below the normal physiological levels.

    Inozyme is preparing to initiate a Phase 1/2 clinical trial in patients with ENPP1 Deficiency in the first half of 2021 and a separate Phase 1/2 clinical trial in patients with ABCC6 Deficiency in mid-2021.

    About Inozyme Pharma

    Inozyme Pharma (NASDAQ:INZY) is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 Deficiencies.

    Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the potential of our lead product candidate, INZ-701, the initiation and timing of our future clinical trials and our research and development programs. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to initiate its planned Phase 1/2 clinical trials of INZ-701 for ENPP1 Deficiency and ABCC6 Deficiency; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties, and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contacts

    Investors:

    Inozyme Pharma

    Stefan Riley, Director of Investor Relations

    Stefan.riley@inozyme.com

    Media:

    SmithSolve

    Alex Van Rees

    (973) 442-1555 ext. 111

    Alex.vanrees@smithsolve.com

     



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  7. – Filed Clinical Trial Application for INZ-701 for ABCC6 Deficiency in Europe; on track to initiate Phase 1/2 clinical trial in mid-2021 –

    – Published peer-reviewed preclinical data supporting INZ-701 as a potential treatment for ENPP1 Deficiency in Journal of Bone and Mineral Research

    – Presented data from first-ever Burden of Illness Study for ENPP1 and ABCC6 Deficiencies at multiple medical conferences –

    – Cash, cash equivalents, and investments expected to enable continued operations into the fourth quarter of 2022 –

    BOSTON, May 12, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today reported financial…

    – Filed Clinical Trial Application for INZ-701 for ABCC6 Deficiency in Europe; on track to initiate Phase 1/2 clinical trial in mid-2021 –

    – Published peer-reviewed preclinical data supporting INZ-701 as a potential treatment for ENPP1 Deficiency in Journal of Bone and Mineral Research

    – Presented data from first-ever Burden of Illness Study for ENPP1 and ABCC6 Deficiencies at multiple medical conferences –

    – Cash, cash equivalents, and investments expected to enable continued operations into the fourth quarter of 2022 –

    BOSTON, May 12, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today reported financial results for the first quarter ended March 31, 2021 and provided recent business highlights.

    "We closed 2020 with the clearance of our IND for INZ-701 by the FDA and our CTA by the MHRA of the UK, both for ENPP1 Deficiency. Since then, we have continued to execute on our plan to deliver a potential therapeutic option for patients with ENPP1 and ABCC6 Deficiencies," said Axel Bolte, MSc, MBA, co-founder, president, and chief executive officer of Inozyme Pharma. "We recently reported key findings from the Burden of Illness Study in patients with both ENPP1 Deficiency and ABCC6 Deficiency. Notably, we found patients in different age groups are impacted by these deficiencies in distinct ways, illuminating an age-based shift that reflects the progression of these debilitating genetic diseases. We expect site activation in the United States for our Phase 1/2 clinical trial site for ENPP1 Deficiency in June 2021 and enrollment of the first patient shortly thereafter. Subject to receiving regulatory clearance, we also expect to initiate our planned Phase 1/2 clinical trial of INZ-701 for ABCC6 Deficiency in mid-2021."

    Mr. Bolte continued, "Our current cash position allows us to appropriately resource each of the key functions necessary to execute on our goals, and is expected to enable our operations into the fourth quarter of 2022."

    Recent Business Highlights

    • Filed Clinical Trial Application for INZ-701 for ABCC6 Deficiency in Europe – The Company expects to initiate a Phase 1/2 clinical trial in mid-2021 and to provide preliminary safety and biomarker data by the end of 2021.
    • Published data supporting INZ-701 as a potential treatment for ENPP1 Deficiency – Peer-reviewed article in the Journal of Bone and Mineral Research shows INZ-701 increased plasma pyrophosphate (PPi) levels, improved disease markers, and decreased mortality in an Enpp1-deficient mouse model. Preclinical findings support increase of PPi levels as a predictive marker of therapeutic benefit.
    • Data presented on utility of INZ-701 as a potential treatment for ABCC6 Deficiency – The Company and research collaborators from Thomas Jefferson University presented data from a preclinical study examining INZ-701 for the potential treatment for ABCC6 Deficiency/Pseudoxanthoma elasticum (PXE) at multiple medical conferences.
    • Data presented from Burden of Illness Study for ENPP1 and ABCC6 Deficiencies – The Company presented data from the first-ever burden of illness study in patients with ENPP1 and ABCC6 Deficiencies at multiple medical conferences.

    Upcoming Anticipated Milestones

    The Company also announced the following anticipated milestones for the INZ-701 clinical development program, subject to COVID-19-related restrictions:

    • ENPP1 Deficiency
      • June 2021: Site activation for Phase 1/2 clinical trial
      • Mid-2021: Enrollment for Phase 1/2 clinical trial
      • Mid-2021: Initiate prospective natural history study
      • H2 2021: Report preliminary safety and biomarker data from Phase 1/2 clinical trial
    • ABCC6 Deficiency
      • Mid-2021: Site activation and enrollment for Phase 1/2 clinical trial
      • By the End of 2021: Report preliminary safety and biomarker data from Phase 1/2 clinical trial

    Financial Results for the Quarter Ended March 31, 2021

    • Cash Position and Financial Guidance – Cash, cash equivalents, and investments were $147.6 million as of March 31, 2021. Based on its current plans, the Company expects that its existing cash, cash equivalents, and investments will be sufficient to enable funding of its operating expenses and capital expenditure requirements into the fourth quarter of 2022.
    • Research and Development (R&D) Expenses – R&D expenses were $6.6 million for the quarter ended March 31, 2021, compared to $6.4 million for the quarter ended March 31, 2020. The increase was primarily due to costs associated with preclinical studies and clinical preparation activities with the Company's contract research organization and increased salaries and employee-related costs due to the growth in the number of R&D employees.
    • General and Administrative (G&A) Expenses – G&A expenses were $4.4 million for the quarter ended March 31, 2021, compared to $1.5 million for the quarter ended March 31, 2020. The increase was primarily due to the growth in the number of G&A employees, an increase in legal fees related to new contracts and operations as a public company and generally higher fees in areas such as audit, tax, and information technology to support the Company's growth.
    • Net Loss – Net loss was $11.1 million, or $0.47 loss per share, for the quarter ended March 31, 2021, compared to $7.7 million, or $6.42 loss per share, for the quarter ended March 31, 2020.

    About Inozyme Pharma

    Inozyme Pharma, Inc. (NASDAQ:INZY), is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 Deficiencies.

    Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation and timing of our clinical trials, the initiation and timing of our natural history study, our research and development programs, the availability of preclinical study and clinical trial data, the timing of our regulatory applications and the period over which we believe that our existing cash, cash equivalents and investments will be sufficient to fund our operating expenses. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to initiate its planned Phase 1/2 clinical trials of INZ-701 for ENPP1 Deficiency and ABCC6 Deficiency; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section in the Company's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Condensed Consolidated Balance Sheet Data

    (Unaudited)

    (in thousands)

      March 31, 2021 December 31, 2020
    Cash, cash equivalents and investments $147,634  $159,896 
    Total assets  158,915   169,363 
    Total liabilities  10,026   11,260 
    Additional paid-in-capital  251,001   249,175 
    Accumulated deficit  (102,126)  (91,076)
    Total stockholders' equity  148,889   158,103 

    Condensed Consolidated Statements of Operations and Comprehensive Loss

    (Unaudited)

    (in thousands, except share and per share data)

      Three Months Ended March 31,
       2021   2020 
    Operating expenses:    
    Research and development $6,603  $6,406 
    General and administrative  4,369   1,500 
    Total operating expenses  10,972   7,906 
    Loss from operations  (10,972)  (7,906)
    Other income (expense):    
    Interest income  63   171 
    Other expenses  (141)  (3)
    Other income (expense), net  (78)  168 
    Net loss $(11,050) $(7,738)
    Other comprehensive income:    
    Unrealized gains on available-for-sale securities  10   23 
    Total other comprehensive income  10   23 
    Comprehensive loss $(11,040) $(7,715)
    Net loss attributable to common stockholders—basic and diluted $(11,050) $(7,738)
    Net loss per share attributable to common stockholders—basic and diluted $(0.47) $(6.42)
    Weighted-average common shares outstanding—basic and diluted  23,429,507   1,205,346 



    Contacts

    Investors:

    Inozyme Pharma

    Stefan Riley, Director of Investor Relations

    Stefan.riley@inozyme.com

    Media:

    Alex Van Rees, SmithSolve

    (973) 442-1555 ext. 111

    Alex.vanrees@smithsolve.com

     



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  8. BOSTON, May 07, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today presented preclinical data suggesting the utility of its lead clinical development candidate, INZ-701, as a potential treatment for ABCC6 Deficiency. The data, presented at the virtual European Calcified Tissue Society Annual Congress (ECTS, May 6-8), are the first to show that an enzyme replacement therapy (ERT) increased plasma pyrophosphate (PPi) levels and reduced calcification in an animal model of ABCC6 Deficiency.

    ABCC6 Deficiency is a rare, inherited disorder that can present as generalized arterial calcification of infancy (GACI) type…

    BOSTON, May 07, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today presented preclinical data suggesting the utility of its lead clinical development candidate, INZ-701, as a potential treatment for ABCC6 Deficiency. The data, presented at the virtual European Calcified Tissue Society Annual Congress (ECTS, May 6-8), are the first to show that an enzyme replacement therapy (ERT) increased plasma pyrophosphate (PPi) levels and reduced calcification in an animal model of ABCC6 Deficiency.

    ABCC6 Deficiency is a rare, inherited disorder that can present as generalized arterial calcification of infancy (GACI) type 2 in infants and as pseudoxanthoma elasticum (PXE) in children and adults. This is one of several disorders with significant decrease in plasma PPi levels, a potent regulator of mineralization. In patients with ABCC6 Deficiency, the abnormal calcification caused by low PPi can result in vision loss and life-threatening cardiovascular complications, among other morbidities. There is no approved treatment for ABCC6 Deficiency.

    "In patients with ABCC6 Deficiency, the reduced levels of PPi that lead to pathological mineralization suggest an overlap between ENPP1 and ABCC6 Deficiencies," explained Yves Sabbagh, Ph.D., Senior Vice President and Chief Scientific Officer of Inozyme Pharma. "This supports the rationale for an enzyme replacement therapy aimed at raising PPi to treat these serious genetic disorders. The data show that INZ-701 increased plasma PPi levels and prevented abnormal calcification in an ABCC6-deficient mouse model, demonstrating its potential for treating patients with PXE, a chronic form of ABCC6 Deficiency with no approved therapeutic options."

    This study was performed in collaboration with Thomas Jefferson University. Subcutaneous administration of INZ-701 (2 and 10 mg/kg every other day for two or eight weeks) was initiated in ABCC6-deficient mice at five to six weeks of age, the time where initiation of ectopic mineralization in this model is observed. INZ-701 led to a dose-dependent increase in plasma PPi levels at both two and eight weeks after initiation of treatment, leading to significantly lower levels of soft tissue mineralization. Histopathologic examination of tissue biopsies from vehicle-treated mice revealed extensive mineralization in the muzzle skin containing vibrissae, a biomarker of the mineralization process in this model. Compared to vehicle-treated mice, a quantitative calcium assay demonstrated that the amount of calcium in muzzle skin biopsies was reduced by 68% and 74% in mice receiving INZ-701 at dose levels of 2 and 10 mg/kg, respectively (p < 0.01).

    "It is encouraging to see an ENPP1 enzyme replacement having an effect on tissue calcification in this PXE animal model. The patients suffering from this disease currently have no treatment options and collaborating with Inozyme to use our in-house expertise on this disease with their drug discovery efforts is exciting," said Jouni Uitto, M.D., Ph.D., Professor and Chair of Dermatology and Cutaneous Biology, and Biochemistry and Molecular Biology, at Thomas Jefferson University. "This study will help Inozyme further characterize the therapeutic potential of INZ-701 in PXE and other manifestations of ABCC6 Deficiency, which may offer hope to patient communities that have been waiting many years for a viable treatment option."

    About ABCC6 Deficiency and Pseudoxanthoma Elasticum (PXE)

    ABCC6 Deficiency is a rare, inherited disorder caused by mutations in the ABCC6 gene, resulting in decreased or absent activity of the ABCC6 protein. A systemic and progressively debilitating condition estimated to affect more than 67,000 individuals worldwide, ABCC6 Deficiency leads to low levels of pyrophosphate (PPi) and is associated with pathological mineralization in blood vessels and soft tissues throughout the body. These effects can result in devastating medical problems including blindness, life-threatening cardiovascular complications, and skin calcification.

    Some infants with ABCC6 Deficiency are diagnosed with generalized arterial calcification of infancy (GACI) type 2, a vascular condition that resembles GACI type 1, the acute infantile form of ENPP1 Deficiency. In older patients, ABCC6 Deficiency presents as pseudoxanthoma elasticum (PXE), a rare, inherited disorder in which individuals develop calcification of soft connective tissues, including in the eyes, cardiovascular system, and skin. Individuals with PXE often have abnormalities in the eyes, such as changes in the pigmented cells of the retina; angioid streaks (tiny cracks in Bruch's membrane, the inner layer of the retina); and choroidal vascularization (bleeding and scarring of the retina), possibly leading to vision loss. Patients with PXE may also exhibit yellowish bumps, papules, on the neck, underarms, and other areas of the skin which becomes leathery and sagging. The skin findings indicate a general, systemic, pathological process of soft tissue calcification.

    About INZ-701

    INZ-701 is an ENPP1 enzyme replacement therapy in development for the treatment of mineralization disorders of the circulatory system, bones, and kidneys. In preclinical studies, the experimental therapy has shown potential to generate plasma pyrophosphate (PPi) and to restore it to appropriate physiological levels, thereby preventing calcification in the vasculature and kidneys, while at the same time correcting bone abnormalities. Inozyme is developing INZ-701 for certain rare, life-threatening, and devastating genetic disorders such as ENPP1 Deficiency and ABCC6 Deficiency in which PPi levels are below the normal physiological levels.

    Inozyme is preparing to initiate a Phase 1/2 clinical trial in patients with ENPP1 Deficiency in the first half of 2021 and a separate Phase 1/2 clinical trial in patients with ABCC6 Deficiency in mid-2021.

    About Inozyme Pharma

    Inozyme Pharma (NASDAQ:INZY) is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 Deficiencies.

    Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the potential of our lead product candidate, INZ-701, the initiation and timing of our future clinical trials and our research and development programs. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to initiate its planned Phase 1/2 clinical trials of INZ-701 for ENPP1 Deficiency and ABCC6 Deficiency; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties, and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contacts

    Investors:

    Inozyme Pharma

    Stefan Riley, Director of Investor Relations

    Stefan.riley@inozyme.com

    Media:

    SmithSolve

    Alex Van Rees

    (973) 442-1555 ext. 111

    Alex.vanrees@smithsolve.com



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  9. BOSTON, May 06, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc., a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced that its co-founder, president, and CEO, Axel Bolte, MSc, MBA, will participate in a fireside chat at the BofA Securities 2021 Virtual Healthcare Conference on Thursday, May 13, at 11:45 a.m. ET.

    The fireside chat will be webcast live and can be accessed from the Investor & News section of Inozyme's website under Events. An archived replay of the webcast will be available for up to 90 days following the event.

    About Inozyme Pharma
    Inozyme Pharma (NASDAQ:INZY) is a rare disease biopharmaceutical company developing novel therapeutics for the…

    BOSTON, May 06, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc., a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced that its co-founder, president, and CEO, Axel Bolte, MSc, MBA, will participate in a fireside chat at the BofA Securities 2021 Virtual Healthcare Conference on Thursday, May 13, at 11:45 a.m. ET.

    The fireside chat will be webcast live and can be accessed from the Investor & News section of Inozyme's website under Events. An archived replay of the webcast will be available for up to 90 days following the event.

    About Inozyme Pharma

    Inozyme Pharma (NASDAQ:INZY) is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 Deficiencies.

    Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

    Contacts

    Investors:

    Inozyme Pharma

    Stefan Riley, Director of Investor Relations

    Stefan.riley@inozyme.com

    Media:

    SmithSolve

    Alex Van Rees

    (973) 442-1555 ext. 111

    Alex.vanrees@smithsolve.com



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  10. - Peer-reviewed article in Journal of Bone and Mineral Research showed INZ-701 increased pyrophosphate (PPi) levels, improved disease markers, and decreased mortality in an ENPP1-deficient mouse model -

    - Preclinical findings support increase of PPi levels as a predictive marker of therapeutic benefit -

    BOSTON, April 29, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc., a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced the online pre-publication release of preclinical data suggesting the potential of its lead development candidate, INZ-701, as a treatment for ENPP1 Deficiency. As reported in the Journal of Bone and Mineral Research (JBMR), in an article titled…

    - Peer-reviewed article in Journal of Bone and Mineral Research showed INZ-701 increased pyrophosphate (PPi) levels, improved disease markers, and decreased mortality in an ENPP1-deficient mouse model -

    - Preclinical findings support increase of PPi levels as a predictive marker of therapeutic benefit -

    BOSTON, April 29, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc., a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced the online pre-publication release of preclinical data suggesting the potential of its lead development candidate, INZ-701, as a treatment for ENPP1 Deficiency. As reported in the Journal of Bone and Mineral Research (JBMR), in an article titled, "INZ‐701 prevents ectopic tissue calcification and restores bone architecture and growth in ENPP1 deficient mice", treatment with INZ-701 was associated with increased plasma levels of pyrophosphate (PPi), a potent regulator of mineralization, as well as improvements in several other disease markers and decreased mortality in a mouse model of ENPP1 Deficiency. The publication is the first to present data on INZ-701 in a peer-reviewed journal.

    "In addition to boosting circulating levels of PPi, INZ-701 prevented pathological calcification in all tested organs, improved growth parameters, corrected bone defects, improved clinical signs, and decreased mortality in ENPP1-deficient mice. These results strengthen the rationale for INZ-701 as a potential treatment for ENPP1 Deficiency and support the endpoints for our planned clinical trials," said Yves Sabbagh, Ph.D., Senior Vice President and Chief Scientific Officer of Inozyme Pharma. "We are pleased that the editors of the JBMR recognize the importance of these preclinical data and their potential implications for patients and families affected by ENPP1 Deficiency."

    ENPP1 Deficiency is a progressive condition that manifests as a spectrum of disease. The most extreme manifestation seen in newborns and infants, generalized arterial calcification of infancy (GACI), is characterized by severe vascular calcification and neointimal proliferation (overgrowth of smooth muscle cells inside blood vessels), resulting in dysfunction and failure of major organs such as the heart and kidneys. The condition is lethal in an estimated 50% of affected babies. Children and adults with ENPP1 Deficiency are affected by autosomal-recessive hypophosphatemic rickets type 2 (ARHR2), which is characterized by bone defects such as rickets and osteomalacia (softened bones), and often exhibit a range of signs and symptoms that can include hearing loss, arterial calcification, cardiac and neurological involvement.

    The JBMR publication highlights the following preclinical findings with INZ-701:

    • Increase of PPi:
      • Durable increases in PPi levels were observed after subcutaneous administration of a single dose of INZ-701 (5 mg/kg) in ENPP1-deficient mice.
      • In a dose-response study, animals injected with INZ-701 (0.2, 1, or 5 mg/kg every other day for eight weeks) showed a dose-dependent elevation in plasma ENPP1 activity and an average increase of approximately 2 µM in plasma PPi. Those findings suggested that INZ-701 at a dose as low as 0.2 mg/kg every other day was sufficient to maintain increased plasma PPi levels after eight weeks.
    • Prevention of calcification:
      • Repeated dosing of INZ-701 at 0.2 mg/kg tended to reduce tissue calcium levels in the kidney, spleen, lung, and liver.
      • Dosing at 1 mg/kg significantly reduced calcification in most of the tissues.
      • Dosing at 5 mg/kg completely prevented calcification in all tissues.
    • Correction of bone defects:
      • INZ-701 treatment led to a dose-dependent increase in trabecular number (a measure of bone texture correlated with bone microarchitecture), cortical thickness, bone volume, and bone mineral density.
    • Restoration of growth parameters:
      • INZ-701 treatment resulted in a clear dose response in rescuing slow growth in ENPP1-deficient mice during the first four weeks of the study.
      • By the end of the study (day 56), mice treated with INZ-701 gained significant weight and reached approximately 15-16 grams in the 0.2 and 1 mg/kg dosing groups, and approximately 18 grams in the 5 mg/kg dosing group, compared to approximately 20 grams in wild-type (WT) mice.
      • ENPP1-deficient mice dosed with vehicle failed to gain weight from roughly four weeks of age to the end of the study.
    • Improvements in other clinical signs:
      • Compared to the vehicle-treated group, ENPP1-deficient mice dosed with 0.2 mg/kg of INZ-701 showed less severe clinical signs associated with ENPP1 deficiency (dehydration, hunched back, stilted gait, rough hair coat and pinned ears) from day 27.
      • Mice dosed with 1 mg/kg of INZ-701 did not show any signs of morbidity until the eighth week of the study.
      • No abnormalities were observed in any WT mice or in ENPP1-deficient mice treated with 5 mg/kg of INZ-701.

    About ENPP1 Deficiency

    The ENPP1 gene produces a critical enzyme called ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1), which regulates inorganic pyrophosphate (PPi) levels in plasma. PPi is essential for preventing harmful soft tissue calcification and for regulating normal bone mineralization. ENPP1 Deficiency affects patients across the age spectrum and manifests as either generalized arterial calcification of infancy (GACI) type 1 or autosomal recessive hypophosphatemic rickets type 2 (ARHR2). GACI type 1 is a devastating and often fatal disease affecting infants and is characterized by calcification and narrowing of large and medium-sized arteries, resulting in heart failure and death in about half of patients within the first six months of life. Mutations in the ABCC6 gene can also cause an infantile onset of the disease called GACI type 2. ARHR2 manifests in the post-infancy stage and causes rickets, weakened bones, repeated bone fractures, skeletal deformities, short stature, muscle weakness, fatigue, and bone pain.

    About INZ-701

    INZ-701 is an enzyme replacement therapy in development for the treatment of mineralization disorders of the circulatory system, bones, and kidneys. In preclinical studies, the experimental therapy has shown potential to generate plasma pyrophosphate (PPi) and to restore it to appropriate physiological levels, thereby preventing calcification in the vasculature and kidneys, while at the same time normalizing bone mineralization. Inozyme is developing INZ-701 for certain rare, life-threatening, and devastating genetic disorders such as ENPP1 Deficiency and ABCC6 Deficiency in which PPi levels are below the normal physiological levels.

    Inozyme is preparing to initiate a Phase 1/2 clinical trial in patients with ENPP1 Deficiency in the first half of 2021 and a separate Phase 1/2 clinical trial in patients with ABCC6 Deficiency in mid-2021.

    About Inozyme Pharma

    Inozyme Pharma (NASDAQ:INZY) is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 Deficiencies.

    Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the potential of our lead product candidate, INZ-701, the initiation and timing of our future clinical trials and our research and development programs. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to initiate its planned Phase 1/2 clinical trials of INZ-701 for ENPP1 Deficiency and ABCC6 Deficiency; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contacts

    Investors:

    Inozyme Pharma

    Stefan Riley, Director of Investor Relations

    Stefan.riley@inozyme.com

    Media:

    SmithSolve

    Alex Van Rees

    (973) 442-1555 ext. 111

    Alex.vanrees@smithsolve.com 



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  11. BOSTON, April 28, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization disorders, today announced that preclinical data from a study examining INZ-701 for the potential treatment for ABCC6 Deficiency/Pseudoxanthoma elasticum (PXE) will be presented at the following upcoming medical conferences:

    Conference: Society for Investigative Dermatology (SID) 2021 Virtual Meeting
    Title: INZ-701 prevents ectopic mineralization in an Abcc6-/- mouse model of pseudoxanthoma elasticum
    Presenter: Joely D. Jacobs, Research Assistant, Department of Dermatology and Cutaneous Biology, Thomas Jefferson University
    Session: Interactive Poster/Exhibitor…

    BOSTON, April 28, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization disorders, today announced that preclinical data from a study examining INZ-701 for the potential treatment for ABCC6 Deficiency/Pseudoxanthoma elasticum (PXE) will be presented at the following upcoming medical conferences:

    Conference: Society for Investigative Dermatology (SID) 2021 Virtual Meeting

    Title: INZ-701 prevents ectopic mineralization in an Abcc6-/- mouse model of pseudoxanthoma elasticum

    Presenter: Joely D. Jacobs, Research Assistant, Department of Dermatology and Cutaneous Biology, Thomas Jefferson University

    Session: Interactive Poster/Exhibitor Session I

    Date and Time: Wednesday, May 5, 2021 from 2:30-4:00 p.m. E.T.

    Conference: European Calcified Tissue Society (ECTS) 2021 Digital Congress

    Title: INZ-701, a recombinant ENPP1-Fc protein, prevents ectopic mineralization in a mouse model of Pseudoxanthoma Elasticum

    Presenter: Zhiliang Cheng, Ph.D., Vice President, Research at Inozyme Pharma

    Session: Plenary Oral Presentations 1: Genetics & Bone

    Date and Time: Friday, May 7, 2021 from 10:30-11:30 a.m. C.E.T.

    About Inozyme Pharma

    Inozyme Pharma (NASDAQ:INZY) is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 Deficiencies.

    Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation and timing of our future clinical trials and our research and development programs. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties, and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contacts

    Investors:

    Inozyme Pharma

    Stefan Riley, Director of Investor Relations

    Stefan.riley@inozyme.com

    Media:

    SmithSolve

    Alex Van Rees

    (973) 442-1555 ext. 111

    Alex.vanrees@smithsolve.com



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  12. BOSTON, April 14, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc., a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization disorders, today presented data that highlight the burden of disease for patients and families affected by ENPP1 Deficiency and ABCC6 Deficiency, two devastating and potentially deadly genetic diseases. In a poster entitled, From the Voice of Patients and Caregivers: Burden of Illness in Infantile Onset ABCC6 and ENPP1 Deficiency (GACI and ARHR2), Inozyme and GACI Global reported that patients in different age groups are impacted in different ways, an age-based shift that reflects the progression of these rare genetic diseases. The poster was presented virtually…

    BOSTON, April 14, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc., a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization disorders, today presented data that highlight the burden of disease for patients and families affected by ENPP1 Deficiency and ABCC6 Deficiency, two devastating and potentially deadly genetic diseases. In a poster entitled, From the Voice of Patients and Caregivers: Burden of Illness in Infantile Onset ABCC6 and ENPP1 Deficiency (GACI and ARHR2), Inozyme and GACI Global reported that patients in different age groups are impacted in different ways, an age-based shift that reflects the progression of these rare genetic diseases. The poster was presented virtually beginning on April 14th at the annual meeting of the American College of Medical Genetics and Genomics (ACMG).

    Patients with ENPP1 Deficiency or ABCC6 Deficiency exhibit a range of signs and symptoms that can include arterial calcification, cardiac and neurological involvement, skeletal abnormalities, and hearing loss. ENPP1 Deficiency manifests as generalized arterial calcification of infancy (GACI) type 1 in infants and autosomal recessive hypophosphatemic rickets type 2 (ARHR2) in children and adults. ABCC6 Deficiency is a disease that can lead to an acute form called GACI type 2 in infants and pseudoxanthoma elasticum (PXE) in older patients.

    "This study is the first to describe the burden of illness in infantile-onset ABCC6 Deficiency and across the age spectrum in patients with ENPP1 Deficiency," noted Catherine Nester, Vice President of Physician and Patient Strategies at Inozyme, and one of the authors of the poster presented at ACMG. "Although we knew that the cardiovascular and skeletal complications of these diseases exact a significant burden on patients and families, we were surprised to learn just how burdensome the actual care coordination can be, from securing a diagnosis, to dealing with multiple tests and treatment options, to managing ongoing care from multiple specialists. The knowledge gained from this study will inform our efforts to develop therapies that we hope will ease the burden for patients and families living with these devastating rare genetic diseases."

    Inozyme worked in partnership with GACI Global to conduct the From the Voice of Patients and Caregivers study. GACI Global is a patient advocacy organization dedicated to providing hope and education to patients and families affected by GACI and ARHR2. The study collected primary patient-reported outcomes data to determine the disease burden in individuals diagnosed with infantile-onset ABCC6 Deficiency or ENPP1 Deficiency in patients of all ages. A total of 38 respondents from nine countries participated in the study, including six individuals with ABCC6 Deficiency, 12 infants with ENPP1 Deficiency, 13 children with ENPP1 Deficiency, and seven adults with ENPP1 Deficiency. Parents or caregivers responded on behalf of patients younger than 18 years of age. The study included responses from parents or caregivers of 11 deceased patients, 10 of whom died within the first 12 months of life.

    The most frequently reported burdens for patients with ENPP1 Deficiency at all time points were:

    • bone and joint pain (100% of adult patients, 85% of pediatric patients)
    • cardiac issues (86% of adult patients, 85% of pediatric patients)
    • mobility issues/fatigue (86% of adult patients, 85% of pediatric patients)

    The most frequently reported symptoms for patients with ABCC6 Deficiency were:

    • gastrointestinal issues (83%)
    • growth and development issues (83%)
    • cardiac issues (67%)

    The study also assessed the importance of each burden for each cohort using a weighted score approach:

    • In the ABCC6 Deficiency cohort, fear of the unknown was the heaviest burden, followed by cardiac issues and difficulty with the hospital experience.
    • In the infant ENPP1 Deficiency cohort, cardiac issues were the greatest burden, followed by difficulty with the hospital experience and issues related to growth and development.
    • In the pediatric ENPP1 Deficiency cohort, treatments/medications were most burdensome, followed by issues related to hearing loss and stress/anxiety.
    • The adult ENPP1 Deficiency cohort was most burdened by issues related to bone/joint pain. Other heavily weighted burdens in this cohort included mobility issues, fatigue, and fear of the unknown.

    "From the unthinkable devastation of a new parent losing their child, to cardiac complications for infants, to the complex medical management of cardiovascular and skeletal issues in pediatric patients, and the cumulative impact of cardiovascular and skeletal complications in adults, this study clearly shows that ENPP1 and ABCC6 Deficiencies are chronic and highly morbid diseases that affect patients of all ages, as reflected in the constellation of physical, emotional, and social burdens across the age continuum," commented Christine O'Brien, co-president of GACI Global and co-author of the study. "We hope our results spur further study of ABCC6 and ENPP1 Deficiencies, and that improved understanding, diagnosis, and management of these rare genetic diseases will alleviate some of the uncertainty and fear for patients and families."

    About ENPP1 Deficiency

    The ENPP1 gene produces a critical enzyme called ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1), which regulates inorganic pyrophosphate (PPi) levels in plasma. PPi is essential for preventing harmful soft tissue calcification and for regulating normal bone mineralization. ENPP1 Deficiency affects patients across the age spectrum and manifests as either generalized arterial calcification of infancy (GACI) type 1 or autosomal recessive hypophosphatemic rickets type 2 (ARHR2). GACI type 1 is a devastating and often fatal disease affecting infants and is characterized by calcification and narrowing of large and medium-sized arteries, resulting in heart failure and death in about half of patients within the first six months of life. Mutations in the ABCC6 gene can also cause an infantile onset of the disease called GACI type 2. ARHR2 manifests in the post-infancy stage and causes rickets, weakened bones, repeated bone fractures, skeletal deformities, short stature, muscle weakness, fatigue, and bone pain.

    About ABCC6 Deficiency

    The ABCC6 gene encodes a protein called ATP-binding cassette sub-family C member 6, a member of the superfamily of ATP-binding cassette (ABC) transporters. ABC proteins transport various molecules across cellular membranes both within and outside of cells. ABCC6 Deficiency is a rare, inherited disorder caused by mutations in the ABCC6 gene, resulting in decreased or absent activity of the ABCC6 protein. A systemic and progressively debilitating condition estimated to affect more than 67,000 individuals worldwide, ABCC6 Deficiency leads to low levels of pyrophosphate (PPi) and is associated with pathological mineralization in blood vessels and soft tissues throughout the body. These effects can result in devastating medical problems including blindness, life-threatening cardiovascular complications, and skin calcification. Some infants with ABCC6 Deficiency are diagnosed with generalized arterial calcification of infancy (GACI) type 2, a vascular condition that resembles GACI type 1. In older patients, ABCC6 Deficiency presents as pseudoxanthoma elasticum (PXE), a rare, inherited disorder in which individuals develop calcification of soft connective tissues, including in the eyes, cardiovascular system, and skin.

    About INZ-701

    INZ-701 is an enzyme replacement therapy in development for the treatment of mineralization disorders of the circulatory system, bones, and kidneys. In preclinical studies, the experimental therapy has shown potential to generate plasma pyrophosphate (PPi) and to restore it to appropriate physiological levels, thereby preventing calcification in the vasculature and kidneys, while at the same time normalizing bone mineralization. Inozyme is developing INZ-701 for certain rare, life-threatening, and devastating genetic disorders such as ENPP1 Deficiency and ABCC6 Deficiency in which PPi levels are below the normal physiological levels.

    Inozyme is preparing to initiate a Phase 1/2 clinical trial in patients with ENPP1 Deficiency in the first half of 2021 and a separate Phase 1/2 clinical trial in patients with ABCC6 Deficiency in mid-2021.

    About Inozyme Pharma

    Inozyme Pharma (NASDAQ:INZY) is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 Deficiencies.

    Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

    About GACI Global

    GACI Global is a nonprofit patient advocacy group whose mission is to connect families affected by Generalized Arterial Calcification of Infancy of Hypophosphatemic Rickets caused by ENPP1 or ABCC6 deficiencies to each other and to the medical community. The organization strives to provide current educational resources and supports ongoing research. The goal of this 100% volunteer-run organization is to provide not only information about what complications can occur due to ENPP1 and ABCC6 deficiencies, but to provide hope for families impacted by the condition around the world.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation and timing of our future clinical trials, our research and development programs, the availability of preclinical study and clinical trial data, the timing of our regulatory applications and the period over which we believe that our existing cash, cash equivalents and investments will be sufficient to fund our operating expenses. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to initiate its planned Phase 1/2 clinical trials of INZ-701 for ENPP1 Deficiency and ABCC6 Deficiency; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties, and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contacts

    Investors:

    Inozyme Pharma

    Axel Bolte, co-founder, president, and chief executive officer

    ir@inozyme.com

    Media:

    SmithSolve

    Alex Van Rees

    (973) 442-1555 ext. 111

    alex.vanrees@smithsolve.com

     



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  13. BOSTON, April 08, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases of abnormal mineralization, today announced that data from the burden of illness in infantile onset ABCC6 and ENPP1 deficiency study will be presented at the American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting taking place April 13-16, 2021.

    The following poster presentation will be available during the ACMG Annual Clinical Genetics Meeting beginning on Wednesday, April 14, 2021, at 7:00 a.m. ET:

    Title: From the Voice of Patients and Caregivers: Burden of Illness in Infantile Onset ABCC6 and ENPP1 Deficiency (GACI and…

    BOSTON, April 08, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases of abnormal mineralization, today announced that data from the burden of illness in infantile onset ABCC6 and ENPP1 deficiency study will be presented at the American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting taking place April 13-16, 2021.

    The following poster presentation will be available during the ACMG Annual Clinical Genetics Meeting beginning on Wednesday, April 14, 2021, at 7:00 a.m. ET:

    Title: From the Voice of Patients and Caregivers: Burden of Illness in Infantile Onset ABCC6 and ENPP1 Deficiency (GACI and ARHR2)

    Poster #: eP024

    The poster will be accessible from the "Investors and Media" section of the Inozyme website at investors.inozyme.com following the conference.

    About Inozyme Pharma

    Inozyme Pharma, Inc. (NASDAQ:INZY) is a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat ENPP1 and ABCC6 deficiencies. ENPP1 and ABCC6 deficiencies are chronic, systemic, and progressive diseases occurring over the course of a patient's lifetime, starting as early as fetal development and spanning into adulthood. ENPP1 and ABCC6 deficiencies are estimated to occur in approximately one in 200,000 and one in 50,000 births, respectively.

    Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation and timing of our future clinical trials and our research and development programs. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties, and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contacts

    Investors:

    Inozyme

    Axel Bolte, co-founder, president, and chief executive officer

    (857) 330-4345

    ir@inozyme.com

    Media:

    SmithSolve

    Alex Van Rees

    (973) 442-1555 ext. 111

    alex.vanrees@smithsolve.com



    Primary Logo

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  14. BOSTON, April 01, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc., a rare disease biopharmaceutical company developing novel therapeutics for the treatment of disorders of abnormal mineralization, announced today changes to its scientific advisory board (SAB), including the addition of three leading key opinion leaders with specific expertise in the company's lead indications:

    • W Charles O'Neill IV, M.D., Director of the Ultrasonography Program in the Renal Division at Emory University School of Medicine
    • Jouni Uitto, M.D., Ph.D., Professor of Dermatology and Cutaneous Biology, and Biochemistry and Molecular Biology, and Chair of the Department of Dermatology and Cutaneous Biology at The Sidney Kimmel Medical College at Thomas Jefferson University…

    BOSTON, April 01, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc., a rare disease biopharmaceutical company developing novel therapeutics for the treatment of disorders of abnormal mineralization, announced today changes to its scientific advisory board (SAB), including the addition of three leading key opinion leaders with specific expertise in the company's lead indications:

    • W Charles O'Neill IV, M.D., Director of the Ultrasonography Program in the Renal Division at Emory University School of Medicine
    • Jouni Uitto, M.D., Ph.D., Professor of Dermatology and Cutaneous Biology, and Biochemistry and Molecular Biology, and Chair of the Department of Dermatology and Cutaneous Biology at The Sidney Kimmel Medical College at Thomas Jefferson University, in Philadelphia, Pennsylvania
    • Paul B. Yu, M.D., Ph.D., Section Head of Cardiovascular Life Sciences at Brigham and Women's Hospital and Associate Professor of Medicine at Harvard Medical School

    David Thompson, M.A., M.S., Ph.D., a senior adviser to Inozyme, who served as Inozyme's Senior Vice President and Chief Scientific Officer from 2018-2020, will also be joining the SAB.

    Enrique M. De La Cruz, Ph.D., Jon S. Morrow, M.D., Ph.D., and Mark A. Lemmon, Ph.D. are stepping down from the SAB, effective immediately.

    "We are honored to welcome Charles, Jouni, and Paul, to Inozyme, and we are excited to have David accept this new role on our scientific advisory board. Their combined experience in ABCC6 deficiency, neointimal proliferation, and mineralization diseases with low pyrophosphate will be invaluable as we continue to explore the role of ENPP1 in new and intriguing indications," said Yves Sabbagh, Ph.D., Senior Vice President and Chief Scientific Officer of Inozyme Pharma. "Inozyme is deeply thankful to Enrique, Jon, and Mark for their valuable advice and counsel through the early years."

    W Charles O'Neill IV, M.D., is an accomplished physician-scientist in the Renal Division at Emory University School of Medicine where he leads an active basic and translational research program. His current research focuses on the pathophysiology of vascular calcification in renal failure, specifically examining the role of endogenous pyrophosphate in the etiology of vascular calcification.

    Jouni Uitto, M.D., Ph.D., is currently Professor of Dermatology and Cutaneous Biology, and Biochemistry and Molecular Biology, and Chair of the Department of Dermatology and Cutaneous Biology at The Sidney Kimmel Medical College at Thomas Jefferson University, in Philadelphia, Pennsylvania. He is also Director of the Jefferson Institute of Molecular Medicine at Thomas Jefferson University. Dr. Uitto is internationally recognized for his research on connective tissue biochemistry and molecular biology in relation to cutaneous diseases and skin aging with a special interest in Pseudoxanthoma Elasticum (PXE), also known as ABCC6 deficiency.

    Paul B. Yu, M.D., Ph.D., is a physician-scientist at Brigham and Women's Hospital and an Associate Professor of medicine at Harvard Medical School. Dr. Yu's clinical focus areas include cardiovascular disease, pulmonary vascular disease, and cardiovascular disease related to rheumatologic conditions. His research explores how signaling via the bone morphogenetic protein (BMP), activin, growth and differentiation factor (GDF), and TGF-beta signaling pathways regulate the consequences of injury and inflammation in cardiovascular, musculoskeletal, and metabolic diseases.

    The new and expanded SAB and the Clinical Advisory Board (CAB) of Inozyme is as follows:

    SAB:

    Yves Sabbagh, Ph.D., ChairInozyme Pharma (Senior Vice President and Chief Scientific Officer)
    Demetrios Braddock, M.D. Ph.D.Yale University School of Medicine
    Charles O'Neill, M.D.Emory University School of Medicine
    Joseph Schlessinger, Ph.D.Yale University School of Medicine
    Ed Skolnik, M.D.New York University Langone Medical Center
    Robert Terkeltaub, M.D.UC San Diego School of Medicine (UCSD)
    David Thompson, M.A., M.S., Ph.D.Inozyme Pharma (Senior Advisor)
    Jouni Uitto, M.D., Ph.D.The Sidney Kimmel Medical College at Thomas Jefferson University
    Paul B. Yu, M.D., Ph.DBrigham and Women's Hospital and Harvard Medical School

    CAB:

    Michael A. Levine, M.D., ChairThe Children's Hospital of Philadelphia (CHOP)
    Thomas O. Carpenter, M.D.Yale University School of Medicine
    Frank Rutsch, M.D.Münster University Children's Hospital

    About Inozyme Pharma

    Inozyme Pharma, Inc. (NASDAQ:INZY), is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 deficiencies.

    Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation and timing of our future clinical trials and our research and development programs. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties, and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contacts

    Investors:

    Inozyme Pharma

    Axel Bolte, co-founder, president, and chief executive officer

    ir@inozyme.com

    Media:

    SmithSolve

    Alex Van Rees

    (973) 442-1555 ext. 111

    alex.vanrees@smithsolve.com



    Primary Logo

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  15. – Received Orphan Drug Designation by the U.S. Food and Drug Administration for INZ-701 for treatment of ABCC6 deficiency –

    – Expect to initiate Phase 1/2 trials of INZ-701 for ENPP1 deficiency in the first half of the year and ABCC6 deficiency by mid-2021 –

    – Cash, cash equivalents, and investments expected to enable continued operations into second half of 2022 –

    BOSTON, March 25, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization, today reported financial results for the full year ended December 31, 2020 and provided recent business highlights.

    "2020 was a foundational year for Inozyme as we laid…

    – Received Orphan Drug Designation by the U.S. Food and Drug Administration for INZ-701 for treatment of ABCC6 deficiency –

    – Expect to initiate Phase 1/2 trials of INZ-701 for ENPP1 deficiency in the first half of the year and ABCC6 deficiency by mid-2021 –

    – Cash, cash equivalents, and investments expected to enable continued operations into second half of 2022 –

    BOSTON, March 25, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization, today reported financial results for the full year ended December 31, 2020 and provided recent business highlights.

    "2020 was a foundational year for Inozyme as we laid the groundwork to become a clinical stage company focused on the biology of mineralization. We have significantly expanded our team and operational footprint, ensuring we have the resources required to bring INZ-701 - our lead product candidate - into two planned first-in-human clinical studies in patients with ENPP1 deficiency and ABCC6 deficiency," said Axel Bolte, MSc, MBA, co-founder, president, and chief executive officer of Inozyme Pharma. "ENPP1 deficiency is a systemic, progressive, and chronic disease that occurs over a patient's lifetime, starting as early as fetal development, and spanning into adulthood, with devastating and often fatal effect. ABCC6 deficiency is a calcification disorder with an onset in adolescence and progressive worsening and high morbidity."

    Mr. Bolte continued, "Based on discussions with U.S. and European regulatory authorities, we plan to initiate our Phase 1/2 clinical trial in ENPP1 deficiency in the first half of 2021 and our Phase 1/2 clinical trial in ABCC6 deficiency by mid-2021."

    Recent Business Highlights

    • Granted Orphan Drug Designation for INZ-701 for ABCC6 Deficiency – Inozyme recently received orphan drug designation by the U.S. Food and Drug Administration for INZ-701 for the treatment of patients with ABCC6 deficiency.
    • Received Clearance to Proceed in U.S. and U.K. with Phase 1/2 Clinical Trial of INZ-701 for the Treatment of ENPP1 Deficiency – the Company expects to initiate the Phase 1/2 trial in the first half of 2021 and to provide preliminary safety and biomarker data in the second half of 2021.
    • Completed the Burden of Disease Patient Study – this study provides an overview of the burden of disease in patients with ENPP1 deficiency and ABCC6 deficiency. The study has been accepted as poster presentations at three scientific conferences in the second quarter of 2021.
    • Completed the Healthy Volunteer Pyrophosphate (PPi) Study – this study validates the highly sensitive assay that will be used to evaluate INZ-701 in clinical trials. The results of this study will be presented at medical conferences later in the year.
    • Advanced Gene Therapy Research and Development – Inozyme progressed research and preclinical proof-of-concept work in demonstrating the ability to raise PPi levels using a gene therapy approach in animal models.

    Upcoming Anticipated Milestones

    The Company also announced the following anticipated milestones for the INZ-701 clinical development program, subject to COVID-19-related restrictions:

    • ENPP1 Deficiency
      • H1 2021: Initiate prospective natural history study
      • H1 2021: Initiate Phase 1/2 clinical trial
      • H2 2021: Report preliminary safety and biomarker data from Phase 1/2 clinical trial
    • ABCC6 Deficiency
      • H1 2021: File Clinical Trial Applications
      • Mid-2021: Initiate Phase 1/2 clinical trial
      • By the End of 2021: Report preliminary safety and biomarker data from Phase 1/2 clinical trial

    Financial Results for the Year Ended December 31, 2020

    • Cash Position and Financial Guidance – Cash, cash equivalents, and investments were $159.9 million as of December 31, 2020. Based on its current plans, the Company expects that its existing cash, cash equivalents, and investments will be sufficient to enable funding of its operating expenses and capital expenditure requirements into the second half of 2022.
    • Research and Development (R&D) Expenses – R&D expenses were $46.5 million for the year ended December 31, 2020, compared to $16.2 million for the year ended December 31, 2019. The increase was primarily due to an increase of $17.8 million resulting from the non-recurring, non-cash purchase of in-process research and development intellectual property assets from Alexion Pharmaceuticals in exchange for stock of the Company in July 2020; costs associated with preclinical studies and clinical preparation activities with the Company's contract research organization; and growth in the number of R&D employees.
    • General and Administrative (G&A) Expenses – G&A expenses were $10.5 million for the year ended December 31, 2020, compared to $4.6 million for the year ended December 31, 2019. The increase was primarily due to the growth in the number of G&A employees; an increase in legal fees related to patents, new contracts, and operations as a public company; and generally higher fees in areas such as audit, tax, and information technology to support the Company's growth.
    • Net Loss – Net loss was $56.4 million, or $5.11 loss per share, for the year ended December 31, 2020, compared to $19.7 million, or $16.67 loss per share, for the year ended December 31, 2019.

    About Inozyme Pharma

    Inozyme Pharma, Inc. (NASDAQ:INZY), is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 deficiencies.

    Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation and timing of our future clinical trials, our research and development programs, the availability of preclinical study and clinical trial data, the timing of our regulatory applications and the period over which we believe that our existing cash, cash equivalents and investments will be sufficient to fund our operating expenses. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to initiate its planned Phase 1/2 clinical trials of INZ-701 for ENPP1 deficiency and ABCC6 deficiency; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Condensed Consolidated Balance Sheet Data

    (Unaudited)

    (in thousands)

     December 31, 2020 December 31, 2019
    Cash, cash equivalents, and investments$159,896  $47,132 
    Total assets 169,363   47,944 
    Total liabilities 11,260   3,236 
    Convertible preferred stock    77,927 
    Additional paid-in-capital 249,175   1,428 
    Accumulated deficit (91,076)  (34,652)
    Total stockholders' equity (deficit) 158,103   (33,219)

    Condensed Consolidated Statements of Operations and Comprehensive Loss

    (Unaudited)

    (in thousands, except share and per share data)

     Year Ended December 31,
      2020   2019 
    Operating expenses:   
    Research and development$46,493  $16,220 
    General and administrative 10,548   4,586 
    Total operating expenses 57,041   20,806 
    Loss from operations (57,041)  (20,806)
    Other income (expense):   
    Interest income 370   1,106 
    Other income (expense), net 247   (24)
    Other income (expense), net 617   1,082 
    Net loss$(56,424) $(19,724)
    Other comprehensive (loss) income:   
    Unrealized (losses) gains on available-for-sale securities (3)  7 
    Total other comprehensive (loss) income (3)  7 
    Comprehensive loss$(56,427) $(19,717)
    Net loss attributable to common stockholders—basic and diluted$(56,424) $(19,724)
    Net loss per share attributable to common stockholders—basic and diluted$(5.11) $(16.67)
    Weighted-average common shares outstanding—basic and diluted 11,036,500   1,183,147 

    Investors:

    Inozyme Pharma

    Axel Bolte, co-founder, president, and chief executive officer

    ir@inozyme.com

    Media:

    Alex Van Rees, SmithSolve

    (973) 442-1555 ext. 111

    alex.vanrees@smithsolve.com

     



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  16. BOSTON, March 11, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc., a rare disease biopharmaceutical company developing novel therapeutics for the treatment of disorders of abnormal mineralization, announced today that ENPP1 deficiency will be featured on The Balancing Act® as part of the show's recurring "Behind the Mystery of Rare and Genetic Diseases" series to raise awareness for this rare, debilitating, and life-threatening mineralization disease for which there are currently no approved treatment options.

    The episode will feature Janine Hicks, a patient living with ENPP1 deficiency; her parents, Donna and Peter Hicks; and Professor Zulf Mughal, a treating physician and researcher and Consultant in Pediatric Bone Disorders, Royal Manchester…

    BOSTON, March 11, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc., a rare disease biopharmaceutical company developing novel therapeutics for the treatment of disorders of abnormal mineralization, announced today that ENPP1 deficiency will be featured on The Balancing Act® as part of the show's recurring "Behind the Mystery of Rare and Genetic Diseases" series to raise awareness for this rare, debilitating, and life-threatening mineralization disease for which there are currently no approved treatment options.

    The episode will feature Janine Hicks, a patient living with ENPP1 deficiency; her parents, Donna and Peter Hicks; and Professor Zulf Mughal, a treating physician and researcher and Consultant in Pediatric Bone Disorders, Royal Manchester Children's Hospital, Manchester University NHS Foundation Trust. The segment will cover scientific and medical aspects of ENPP1 deficiency, the importance of making an accurate diagnosis, and the daily challenges faced by patients and families living with this debilitating disease.

    "Many rare diseases like ENPP1 deficiency are not easily recognized or well-understood and patients face long delays in getting an accurate diagnosis. It is our hope that this segment will increase awareness of this devastating disease so that patients can receive an accurate diagnosis and appropriate medical treatment as we work to develop a therapy to treat patients who currently lack options," said Axel Bolte, co-founder, president, and chief executive officer of Inozyme. "We are extremely grateful to Janine, Donna, Peter, and Dr. Mughal for telling this powerful story and bringing a voice to the many challenges that patients and families dealing with ENPP1 deficiency face every day."

    "At GACI Global, we provide information about the complications that can occur with ENPP1 and ABCC6 deficiency and seek to bring hope to families impacted by these rare and life-threatening conditions," said Christine O'Brien and Liz Molloy, co-presidents of GACI Global. "Our mission is to raise awareness of these rare conditions through research and education, so we welcomed this opportunity to work with Inozyme on this initiative. We, too, are grateful for everyone's participation in the development of this segment."

    For the past 10 years, Behind the Mystery has spotlighted undiagnosed, misdiagnosed, and complex medical disorders by partnering with pharmaceutical and biotechnology companies who are on a mission to educate the public to bring earlier diagnosis and treatment and provide a sense of community for people living with these disorders.

    The segment will air on The Balancing Act® on Lifetime® on Monday, March 15, 2021 at 7:30 a.m., ET/PT and again on Wednesday, March 24, 2021 at 7:30 a.m. ET/PT, after which it will be shown in syndication.

    About ENPP1 Deficiency

    The ENPP1 gene produces a critical enzyme called ectonucleotide pyrophosphatase/ phosphodiesterase 1 (ENPP1), which regulates inorganic pyrophosphate (PPi) levels in plasma. PPi is essential for preventing harmful soft tissue calcification and for regulating normal bone mineralization. ENPP1 Deficiency affects patients across the age spectrum and manifests as either generalized arterial calcification of infancy (GACI) type 1 or autosomal recessive hypophosphatemic rickets type 2 (ARHR2). GACI type 1 is a devastating and often fatal disease affecting infants and is characterized by calcification and narrowing of large and medium-sized arteries, resulting in heart failure and death in about half of patients within the first six months of life. Mutations in the ABCC6 gene can also cause an infantile onset of the disease called GACI Type 2. ARHR2 manifests in the post-infancy stage and causes rickets, weakened bones, repeated bone fractures, skeletal deformities, short stature, muscle weakness, fatigue, and bone pain.

    About The Balancing Act

    The Balancing Act® is a morning show created and produced by BrandStar that offers sensible solutions and essential information in a fun, entertaining format; providing resources to help people do life better. The Balancing Act features everything from delicious recipes, style makeovers and dream getaways to parenting tips and the latest news in health and wealth. Tune in to The Balancing Act weekdays at 7:30 a.m. (ET/PT) on Lifetime® and find all previously aired episodes on TheBalancingAct.com.

    About GACI Global

    GACI Global is a nonprofit patient advocacy group whose mission is to connect families affected by Generalized Arterial Calcification of Infancy of Hypophosphatemic Rickets caused by ENPP1 or ABCC6 deficiencies to each other and to the medical community. The organization strives to provide current educational resources and supports ongoing research. The goal of this 100% volunteer run organization is to provide not only information about what complications can occur due to ENPP1 and ABCC6 deficiencies, but to provide hope for families impacted by the condition around the world.

    About Inozyme Pharma

    Inozyme Pharma, Inc. (NASDAQ:INZY), is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 deficiencies.

    Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation and timing of our future clinical trials and our research and development programs. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties, and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contacts

    Investors:

    Inozyme Pharma

    Axel Bolte, co-founder, president, and chief executive officer

    ir@inozyme.com

    Media:

    SmithSolve

    Alex Van Rees

    (973) 442-1555 ext. 111

    alex.vanrees@smithsolve.com 



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  17. BOSTON, Feb. 04, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton, today announced the appointment of Deborah Wenkert, M.D. as senior vice president and chief medical officer, effective February 2, 2021.

    Dr. Wenkert, a leading pediatric rheumatologist with more than 20 years of experience in metabolic bone and genetic disorders, will direct Inozyme's clinical development programs as well as scientific communications activities. She succeeds Pedro Huertas, M.D., Ph.D., who has stepped down from his position to pursue other interests.

    "It is a…

    BOSTON, Feb. 04, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton, today announced the appointment of Deborah Wenkert, M.D. as senior vice president and chief medical officer, effective February 2, 2021.

    Dr. Wenkert, a leading pediatric rheumatologist with more than 20 years of experience in metabolic bone and genetic disorders, will direct Inozyme's clinical development programs as well as scientific communications activities. She succeeds Pedro Huertas, M.D., Ph.D., who has stepped down from his position to pursue other interests.

    "It is a great pleasure to welcome Deborah Wenkert to the Inozyme team. Inozyme will benefit from her expertise in developing novel treatments for rare bone disorders as we plan to bring INZ-701 into clinical trials in the first half of 2021. Dr. Wenkert, a pediatrician, has dedicated her career to the care for patients with severe metabolic bone disease. Her experience as a pediatric rheumatologist, researcher, and industry executive will be invaluable as we explore new indications in rare bone disease," said Axel Bolte, M.Sc., M.B.A., co-founder, president and chief executive officer of Inozyme Pharma. "We thank Dr. Pedro Huertas for his contributions to the company and wish him well in his future endeavors."

    Dr. Wenkert previously served as chief medical officer of PreciThera, Inc., where she managed several early clinical development programs in rare bone diseases. Before that, Dr. Wenkert was a medical director in clinical research at Amgen Inc., where she helped design Phase 2 and lead Phase 3 and 4 clinical trials in the inflammation and bone therapeutic areas. Both at Amgen and since, she has facilitated multiple regulatory interactions and submissions.

    Dr. Wenkert's career in academic medicine and clinical research includes more than a decade at St. Louis University School of Medicine, where she was an associate adjunct clinical professor of pediatrics. As associate director of the Center for Metabolic Bone Disease and Molecular Research at Shriners Hospital for Children in St. Louis, Dr. Wenkert conducted research and provided clinical care to children living with metabolic bone and genetic disorders and has volunteered there since.

    "I am excited to join Inozyme's team as we advance the understanding of devastating and deadly diseases of abnormal mineralization," said Dr. Wenkert. "Building on strong scientific and preclinical data, Inozyme's drug development plans are following a clear path to help patients with ENPP1 deficiency as well as ABCC6 deficiency. I look forward to working with the team to achieve these important goals."

    Dr. Wenkert holds a B.A. in Biochemistry from Rice University, and an M.D. from the University of Texas Medical Branch. Dr. Wenkert is board-certified in pediatrics and pediatric rheumatology. She is the author of more than 35 peer-reviewed research publications, more than 100 scientific conference presentations and has authored two book chapters.

    About Inozyme Pharma

    Inozyme Pharma, Inc. (NASDAQ:INZY), is a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat ENPP1 and ABCC6 deficiencies. ENPP1 and ABCC6 deficiencies are chronic, systemic, and progressive diseases occurring over the course of a patient's lifetime, starting as early as fetal development and spanning into adulthood. ENPP1 and ABCC6 deficiencies are estimated to occur in approximately one in 200,000 and one in 50,000 births, respectively.

    Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation and timing of our future clinical trials and our research and development programs. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties, and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contacts

    Investors:

    Brian Luque, Director, Investor Relations

    (951) 206-1200

    ir@inozyme.com

    Media:

    Alex Van Rees, SmithSolve

    (973) 442-1555 ext. 111

    alex.vanrees@smithsolve.com

     



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  18. – U.S. Food and Drug Administration cleared Investigational New Drug Application –

    – United Kingdom Medicines and Healthcare Products Regulatory Agency authorized Clinical Trial Application –

    – Program addressing rare mineralization disorders expected to enroll first subject in H1'21 and provide preliminary safety and biomarker data in H2'21 –

    BOSTON, Jan. 04, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Company's Investigational New Drug (IND) application…

    – U.S. Food and Drug Administration cleared Investigational New Drug Application –

    – United Kingdom Medicines and Healthcare Products Regulatory Agency authorized Clinical Trial Application –

    – Program addressing rare mineralization disorders expected to enroll first subject in H1'21 and provide preliminary safety and biomarker data in H2'21 –

    BOSTON, Jan. 04, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Company's Investigational New Drug (IND) application and that the United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA) has authorized its Clinical Trial Application (CTA) for a Phase 1/2 clinical trial evaluating INZ-701 in adults with ENPP1 deficiency. The Company expects to enroll the first subject in the first half of 2021 and provide preliminary safety and biomarker data in the second half of 2021.

    "With these important regulatory clearances for our first-in-human clinical trial for INZ-701 in subjects with ENPP1 deficiency, we have transitioned from a research-stage to a clinical-stage company. This is a significant milestone in our mission to develop therapeutic breakthroughs in diseases of abnormal mineralization," said Axel Bolte, MSc, MBA, co-founder, president and chief executive officer of Inozyme Pharma. "We are pleased to begin 2021 by ramping up study start up activities and look forward to dosing subjects in the first half of the year."

    About the INZ701-101 Phase 1/2 Clinical Trial of INZ-701 in Adults with ENPP1 Deficiency

    The Phase 1/2 clinical trial is a multi-center, open-label, first-in-human, multiple ascending dose study in adults with ENPP1 deficiency. The trial is expected to enroll nine adult subjects across three dose cohorts with three subjects per cohort. Subjects will participate in a pre-dosing screening period followed by a four-week treatment period in which subjects will receive INZ-701 subcutaneously twice weekly. The Phase 1/2 clinical trial will primarily investigate the safety and tolerability of INZ-701 and characterize its pharmacokinetic and pharmacodynamic profile, including plasma pyrophosphate (PPi) and other biomarker levels, to establish a recommended dosing regimen for further clinical development. Exploratory objectives include obtaining baseline measurements of calcification, patient reported outcomes and quality of life.

    Additional details can be found:

    https://clinicaltrials.gov/ct2/show/NCT04686175

    About INZ-701

    INZ-701 is a soluble, recombinant protein containing the extracellular domain of native human ENPP1 fused to the Fc domain of the immunoglobulin IgG1 that is designed to correct a defect in the mineralization pathway caused by ENPP1 and ABCC6 deficiencies. In preclinical studies conducted in ENPP1-deficient and ABCC6-deficient mouse models, dosing with INZ-701 resulted in normalized levels of PPi and reduced tissue calcification. The FDA has granted orphan drug, rare pediatric disease, and fast track designations to INZ-701 for the treatment of ENPP1 deficiency. The European Medicines Agency has also granted orphan drug designation to INZ-701 for the treatment of ENPP1 deficiency.

    About Inozyme Pharma

    Inozyme Pharma, Inc. (NASDAQ:INZY), is a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat ENPP1 and ABCC6 deficiencies. ENPP1 and ABCC6 deficiencies are chronic, systemic, and progressive diseases occurring over the course of a patient's lifetime, starting as early as fetal development and spanning into adulthood. ENPP1 and ABCC6 deficiencies are estimated to occur in approximately one in 200,000 and one in 50,000 births, respectively.

    Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation and timing of our future clinical trials, our research and development programs, the availability of preclinical study and clinical trial data, and the timing of our regulatory applications. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Investors:

    Brian Luque, Director, Investor Relations

    (951) 206-1200

    ir@inozyme.com

    Media:

    Alex Van Rees, SmithSolve

    (973) 442-1555 ext. 111

    alex.vanrees@smithsolve.com



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  19. –  Submitted CTA for INZ-701 for the treatment of ENPP1 deficiency to United Kingdom regulatory agency –

    –  Received Rare Pediatric Disease and Fast Track Designations for INZ-701 for the treatment of ENPP1 deficiency –

    –  Expect to initiate INZ-701 Phase 1/2 clinical trials for ENPP1 and ABCC6 deficiencies in first half of 2021 –

    BOSTON, Nov. 12, 2020 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton, today reported financial results for the third quarter ended September 30, 2020 and provided recent business highlights.

    "ENPP1 deficiency is a systemic…

    –  Submitted CTA for INZ-701 for the treatment of ENPP1 deficiency to United Kingdom regulatory agency –

    –  Received Rare Pediatric Disease and Fast Track Designations for INZ-701 for the treatment of ENPP1 deficiency –

    –  Expect to initiate INZ-701 Phase 1/2 clinical trials for ENPP1 and ABCC6 deficiencies in first half of 2021 –

    BOSTON, Nov. 12, 2020 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton, today reported financial results for the third quarter ended September 30, 2020 and provided recent business highlights.

    "ENPP1 deficiency is a systemic, progressive and continuous disease occurring over the course of a patient's lifetime, starting as early as fetal development and spanning into adulthood. The fact that INZ-701 had previously received orphan drug designation and now rare pediatric disease and fast track designations underscores the significant unmet medical need for a treatment for this disease," said Axel Bolte, MSc, MBA, co-founder, president and chief executive officer of Inozyme Pharma. "I'm pleased with the progress we have made with U.S. and European regulatory authorities, and we remain on track to initiate our planned Phase 1/2 clinical trials in the first half of 2021, subject to clearance of our regulatory applications."

    Recent Business Highlights

    • Submitted Clinical Trial Application (CTA) for INZ-701 for the treatment of ENPP1 deficiency – Inozyme recently submitted its first CTA to initiate a Phase 1/2 clinical trial of INZ-701 for the treatment of ENPP1 deficiency to the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA).
    • Received Rare Pediatric Disease Designation and Fast Track Designation from the U.S. Food and Drug Administration (FDA) for INZ-701 for the treatment of ENPP1 deficiency – The FDA grants rare pediatric disease designation to drugs for serious and life-threatening diseases in which the serious or life-threatening manifestations primarily affect children aged from birth through 18 years and affect fewer than 200,000 people in the U.S. Under the FDA's Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives approval of a biologics license application (BLA) for a rare pediatric disease product application may be eligible for a voucher which can be redeemed to obtain priority review for a subsequent marketing application for a different product. Separately, Fast Track Designation facilitates the potential expedited development and review of a drug for the treatment of a serious or life-threatening disease and that has demonstrated the potential to address unmet medical needs. Benefits of this designation include frequent engagements with the FDA to discuss the drug's clinical development plan, eligibility for priority review, and a rolling review of a BLA. Previously, the FDA and the European Medicines Agency (EMA) had granted orphan drug designation to INZ-701 for the treatment of ENPP1 deficiency.
    • Completed disease burden study in ENPP1 deficiency and ABCC6 deficiency – Inozyme and GACI Global, a patient advocacy organization dedicated to bettering the lives of families affected by Generalized Arterial Calcification of Infancy and/or Autosomal Recessive Hypophosphatemic Rickets Type 2 (GACI/ARHR2), completed a study to characterize the burden of disease and understand the systemic progression of disease for the rare genetic diseases of both ENPP1 deficiency and ABCC6 deficiency from the perspective of a patient and/or parent. Inozyme expects to share data from this study in 2021.

    Upcoming Anticipated Milestones, Subject to COVID-19 Dynamics

    • INZ-701 for ENPP1 deficiency
      • Early 2021: Clearance of IND and CTAs
      • H1 2021: Initiation of Phase 1/2 clinical trial
      • H1 2021: Initiation of prospective natural history study
      • H2 2021: Preliminary safety and biomarker data from Phase 1/2 clinical trial
    •  INZ-701 for ABCC6 deficiency
      • Early 2021: Clearance of CTAs
      • H1 2021: Initiation of Phase 1/2 clinical trial
      • H2 2021: Preliminary safety and biomarker data from Phase 1/2 clinical trial

    Upcoming Investor Conference

    • Piper Sandler 32nd Annual Healthcare Conference, November 30 – December 3, 2020

    Third Quarter 2020 Financial Results

    • Cash Position and Financial Guidance – Cash, cash equivalents and investments were $171.7 million as of September 30, 2020. Based on its current plans, the Company expects that its existing cash, cash equivalents and investments will be sufficient to enable it to fund its operating expenses and capital expenditure requirements at least into the second half of 2022.
    • Research and Development (R&D) Expenses– R&D expenses were $25.2 million for the third quarter ended September 30, 2020, compared to $3.3 million for the same period in 2019. The increase was primarily due to an increase of $17.8 million resulting from the non-recurring, non-cash purchase of in-process research and development intellectual property assets from Alexion in exchange for stock of the Company in July 2020, costs associated with preclinical studies and clinical preparation activities with the Company's CRO, and growth in the number of R&D employees.
    • General and Administrative (G&A) Expenses – G&A expenses were $3.1 million for the third quarter ended September 30, 2020, compared to $1.0 million for the same period in 2019. The increase was primarily due to the growth in the number of G&A employees, an increase in legal fees related to patents, new contracts and operations as a public company, and generally higher fees in areas such as audit, tax and information technology to support the Company's growth.
    • Net Loss – Net loss was $28.1 million, or $1.55 loss per share, for the third quarter ended September 30, 2020, compared to $4.0 million, or $3.38 loss per share, for the same period in 2019.

    About Inozyme Pharma

    Inozyme Pharma, Inc. (NASDAQ:INZY), is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 deficiencies.

    Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation and timing of our future clinical trials, our research and development programs, the availability of preclinical study and clinical trial data, the timing of our regulatory applications and the period over which we believe that our existing cash, cash equivalents and investments will be sufficient to fund our operating expenses. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to successfully resolve the clinical hold with regard to its planned Phase 1/2 clinical trial of INZ-701 for ENPP1 deficiency; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Condensed Consolidated Balance Sheet Data

    (Unaudited)

    (in thousands)

     September 30,

    2020
     December 31,

    2019
    Cash, cash equivalents and investments$171,709  $47,132 
    Total assets 178,993   47,944 
    Total liabilities 11,077   3,236 
    Convertible preferred stock    77,927 
    Additional paid-in-capital 247,872   1,428 
    Accumulated deficit (79,958)  (34,652)
    Total stockholders' equity (deficit) 167,916   (33,219)
        

    Condensed Consolidated Statements of Operations and Comprehensive Loss

    (Unaudited)

    (in thousands, except share and per share data)

     Three Months Ended September 30, Nine Months Ended September 30,
     2020 2019 2020 2019
    Operating expenses:       
    Research and development$             25,174  $               3,317  $             39,457  $             10,941 
    General and administrative   3,142     1,003     6,313     3,097 
    Total operating expenses   28,316     4,320     45,770     14,038 
    Loss from operations   (28,316)    (4,320)    (45,770)    (14,038)
    Other income (expense):       
    Interest income   64     288     306     892 
    Other income (expense), net   157     (3)    158     (34)
    Other income (expense), net   221     285     464     858 
    Net loss$            (28,095) $              (4,035) $            (45,306) $            (13,180)
    Other comprehensive (loss) income:       
    Unrealized (losses) gains on available-for-sale securities   (13)    (2)    (5)    8 
    Total other comprehensive (loss) income   (13)    (2)    (5)    8 
    Comprehensive loss$            (28,108) $              (4,037) $            (45,311) $            (13,172)
    Net loss attributable to common stockholders—basic and diluted$            (28,095) $              (4,035) $            (45,306) $            (13,180)
    Net loss per share attributable to common stockholders—basic and diluted$                (1.55) $                (3.38) $                (6.57) $              (11.20)
    Weighted-average common shares outstanding—basic and diluted   18,101,496     1,195,309     6,893,745     1,176,769 
            

    Investors:

    Brian Luque, Director, Investor Relations

    (951) 206-1200

    ir@inozyme.com

    Media:

    Alex Van Rees, SmithSolve

    (973) 442-1555 ext. 111

    alex.vanrees@smithsolve.com

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  20. BOSTON, Nov. 09, 2020 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton, today announced the appointment of Kevin B. Johnson, Ph.D., MBA, as senior vice president, regulatory affairs, effective immediately.

    Dr. Johnson brings to Inozyme more than 25 years of experience in developing and implementing global regulatory and clinical development strategies for rare diseases across entire product development lifecycles from preclinical through clinical development and ultimately to product approval for a variety of drugs, biologics, combination products and cell/gene…

    BOSTON, Nov. 09, 2020 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton, today announced the appointment of Kevin B. Johnson, Ph.D., MBA, as senior vice president, regulatory affairs, effective immediately.

    Dr. Johnson brings to Inozyme more than 25 years of experience in developing and implementing global regulatory and clinical development strategies for rare diseases across entire product development lifecycles from preclinical through clinical development and ultimately to product approval for a variety of drugs, biologics, combination products and cell/gene therapies. Dr. Johnson will be responsible for leading Inozyme's global regulatory strategy.

    "We are thrilled to have Kevin join the Inozyme leadership team during this period of steady execution and as we prepare to enter clinical development," said Axel Bolte, MSc, MBA, co-founder, president and chief executive officer of Inozyme Pharma. "Kevin is a talented regulatory affairs executive with deep experience in rare diseases and I look forward to Kevin's contributions in helping Inozyme meet the needs of underserved patient communities."

    Dr. Johnson joins Inozyme from Magenta Therapeutics, Inc., where he served as the senior vice president, head of regulatory and quality, and led global strategy for a portfolio of biologics. Prior to that, he served as senior vice president, head of regulatory affairs, quality and pharmacovigilance at IMARA Inc., during which time the company received several regulatory designations for orphan diseases such as sickle cell disease and beta-thalassemia. Before IMARA, Dr. Johnson led global regulatory strategies at Vtesse (later acquired by Sucampo), addressing ultra-rare diseases such as Niemann-Pick disease type C under Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) and Promising Innovative Medicine designation from the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA). Dr. Johnson also served as the director, global regulatory affairs for rare diseases and gene therapies at GlaxoSmithKline, working on the international regulatory team for the European approval of the gene therapy Strimvelis® for ADA-SCID, and which team subsequently received Regenerative Medicine Advanced Therapy (RMAT) designation for a retinal gene therapy product.

    "Based on the compelling science and preclinical research conducted with INZ-701, Inozyme has the potential to help patients with devastating and debilitating rare metabolic diseases who currently lack effective treatment options," said Dr. Johnson. "I am excited to join Inozyme at such an important time in the company's growth trajectory, and I look forward to contributing to Inozyme's success."

    Dr. Johnson holds a Ph.D. in Neurobiology from the University of North Carolina School of Medicine, an MBA from the Kenan-Flagler School of Business at the University of North Carolina, and a B.S. in Chemistry from the University of South Florida. Dr. Johnson also holds a Regulatory Affairs Certification (RAC) credential from the Regulatory Affairs Professional Society.

    About Inozyme Pharma

    Inozyme Pharma is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 deficiencies.

    Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to our research and development programs. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to successfully resolve the clinical hold with regard to its planned Phase 1/2 clinical trial of INZ-701 for ENPP1 deficiency; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Investors:

    Brian Luque, Director, Investor Relations

    (951) 206-1200

    ir@inozyme.com

    Media:

    Alex Van Rees, SmithSolve

    (973) 442-1555 ext. 111

    alex.vanrees@smithsolve.com

    Primary Logo

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  21. Dr. Sabbagh Brings More Than 20 Years of Experience in Rare Genetic Disorders and Mineral Metabolism Research Development Programs

    Company Announces Retirement of David Thompson, Ph.D.

    – Company to Open New Laboratory Space to Increase R&D Capabilities

    BOSTON, Oct. 13, 2020 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton, today announced the appointment of Yves Sabbagh, Ph.D., as senior vice president and chief scientific officer, and the retirement of David Thompson, Ph.D., Inozyme's former chief scientific officer.

    Dr. Sabbagh brings to Inozyme…

    Dr. Sabbagh Brings More Than 20 Years of Experience in Rare Genetic Disorders and Mineral Metabolism Research Development Programs

    Company Announces Retirement of David Thompson, Ph.D.

    – Company to Open New Laboratory Space to Increase R&D Capabilities

    BOSTON, Oct. 13, 2020 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton, today announced the appointment of Yves Sabbagh, Ph.D., as senior vice president and chief scientific officer, and the retirement of David Thompson, Ph.D., Inozyme's former chief scientific officer.

    Dr. Sabbagh brings to Inozyme more than 20 years of experience in rare genetic disorders and mineral metabolism with responsibilities leading to the identification and evaluation of novel therapeutic approaches and translating them into clinical candidates. Dr. Sabbagh will be responsible for expanding Inozyme's proprietary pipeline by identifying and developing new therapeutics for monogenic and non-genetic diseases of abnormal mineralization.

    "Yves is an accomplished and well-regarded scientist in the areas of renal, bone and mineral research and brings to Inozyme extensive management experience leading teams developing highly innovative drug candidates," said Axel Bolte, MSc, MBA, co-founder, president and chief executive officer of Inozyme Pharma. "I want to express my deep appreciation for David's extensive contributions over the last several years. He has been instrumental in establishing the research group at Inozyme and our portfolio of indications for INZ-701. We look forward to maintaining an active relationship with David in his new role as senior advisor to the Company and wish him the very best in his retirement."

    Prior to joining Inozyme, Dr. Sabbagh served as the head of rare renal and musculoskeletal diseases research at Sanofi. Prior to that executive role, he held scientific roles of increasing responsibility at Sanofi and Genzyme Corporation spanning endocrine, renal and rare bone diseases including driving the strategy for bone indications. Prior to his corporate experience, he was an instructor at the Harvard Medical School in the Endocrine unit.

    "Based on the compelling science and the quality of translational research conducted in mineralization disorders with an initial focus on ENPP1 and ABCC6 deficiencies, Inozyme has the potential to help patients with devastating and debilitating rare diseases that currently lack effective treatment options," said Dr. Sabbagh. "I am excited to be joining Inozyme at such an important time in its growth trajectory. And I look forward to leveraging the company's new lab infrastructure in Boston's innovative Seaport District to contribute to Inozyme's continued success and bring transformative therapies to patients."

    Dr. Sabbagh has co-authored more than 30 peer-reviewed publications and book chapters and is a member of several scientific societies. Dr. Sabbagh received a B.Sc. in biochemistry from McGill University, an MSc in microbiology from Université Laval and a Ph.D. in biology from McGill University.

    In conjunction with Dr. Sabbagh's appointment, Dr. Thompson announced his retirement as senior vice president and chief scientific officer. Dr. Thompson will step down from his role following a transition period and will remain associated with the Company in his role as senior advisor.

    About Inozyme Pharma

    Inozyme Pharma is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of potentially first-in-class therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 deficiencies.

    Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to our research and development programs. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to successfully resolve the clinical hold with regard to its planned Phase 1/2 clinical trial of INZ-701 for ENPP1 deficiency; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Investors:

    Brian Luque, Director, Investor Relations

    (951) 206-1200

    ir@inozyme.com

    Media:

    Alex Van Rees, SmithSolve

    (973) 442-1555 ext. 111

    alex.vanrees@smithsolve.com

    Primary Logo

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  22. - Longitude Venture Partners IV will build on the Firm's commitment to improve clinical outcomes, enhance patient quality of life, and drive efficiency in healthcare delivery -

    - In 2020 to date, Longitude portfolio companies announced 4 IPOs and 3 M&A transactions -

    MENLO PARK, Calif. and GREENWICH, Conn. and BOSTON, Sept. 23, 2020 (GLOBE NEWSWIRE) -- Longitude Capital, a leading healthcare venture capital firm, today announced the closing of Longitude Venture Partners IV, L.P. ("LVP4"), with $585 million in capital commitments. LVP4, the largest fund that Longitude Capital has raised to date, will build on the proven strategy of its predecessor funds by investing in biotechnology, medical technology, and health solutions companies that…

    - Longitude Venture Partners IV will build on the Firm's commitment to improve clinical outcomes, enhance patient quality of life, and drive efficiency in healthcare delivery -

    - In 2020 to date, Longitude portfolio companies announced 4 IPOs and 3 M&A transactions -

    MENLO PARK, Calif. and GREENWICH, Conn. and BOSTON, Sept. 23, 2020 (GLOBE NEWSWIRE) -- Longitude Capital, a leading healthcare venture capital firm, today announced the closing of Longitude Venture Partners IV, L.P. ("LVP4"), with $585 million in capital commitments. LVP4, the largest fund that Longitude Capital has raised to date, will build on the proven strategy of its predecessor funds by investing in biotechnology, medical technology, and health solutions companies that seek to transform the healthcare industry.

    "We are in the golden era of medical and biological innovation. The significant unmet clinical needs and the inefficiencies of our current healthcare system are driving meaningful scientific breakthroughs and creative solutions," said Patrick Enright, co-founder and Managing Director of Longitude Capital. Since inception in 2006, Longitude Capital has raised nearly $2 billion in cumulative capital commitments and managed over 30 company exits. "We are fortunate to have helped build many successful healthcare companies and look forward to working alongside the next wave of entrepreneurs and scientists to advance critical medicines, devices, and health solutions to the marketplace."

    Juliet Bakker, co-founder and Managing Director of Longitude Capital, added, "We are thankful for the continued support of, and partnerships with, our investors who value our differentiated venture growth investing approach, experienced team, and passion for forging new frontiers in healthcare."

    LVP4 will invest opportunistically across all stages of a company's development through a variety of approaches that include traditional venture capital investing and special situations such as spin-outs, equity-linked transactions, and private investments in public equities. Many of these investments stem from Longitude's proprietary research of targeted healthcare sectors, therapeutic areas, and technologies of interest.

    Longitude Capital's recent Initial Public Offerings (IPOs) and exits include 89bio (NASDAQ:ETNB), Aimmune (NASDAQ:AIMT, recently agreed to be acquired by Nestlé Health Science))), Axonics Modulation Technologies (NASDAQ:AXNX), Checkmate Pharmaceuticals (NASDAQ:CMPI), Inflazome (acquired by Roche), Inozyme Pharma (NASDAQ:INZY), KaNDy Therapeutics (acquired by Bayer), Molecular Templates (NASDAQ:MTEM), Poseida Therapeutics (NASDAQ:PSTX), and Vaxcyte (NASDAQ:PCVX).

    About Longitude Capital

    Longitude Capital is a leading healthcare venture capital firm, that invests in transformative biotechnology, medical technology, and health solutions companies seeking to improve clinical outcomes, enhance quality of life, and drive efficiency of healthcare delivery. Founded in 2006, Longitude Capital invests in both privately held and publicly traded companies through a variety of investment approaches. Longitude Capital has offices in Menlo Park, CA, Greenwich, CT, and Boston, MA. For more information, including a complete listing of investments, please visit www.longitudecapital.com.

    About Longitude Capital Recent IPOs and Exits

    • 89bio is a biopharmaceutical company developing and commercializing innovative therapies for the treatment of liver and cardio-metabolic diseases. 89bio was originally spun out of Teva Pharmaceuticals in 2018 by founding investors Longitude Capital and OrbiMed, and completed its IPO in November 2019. www.89bio.com.



    • Aimmune is a biopharmaceutical company developing and commercializing treatments for potentially life-threatening food allergies. Aimmune's PALFORZIA® is the world's first approved treatment for peanut allergy. Longitude Capital was the founding institutional investor of Aimmune. In 2020, Aimmune agreed to be acquired by Nestlé Health Science for $34.50 per share in cash, representing a total equity value of $2.6 billion. www.aimmune.com.



    • Axonics Modulation Technologies is a medical technology company developing and commercializing novel implantable rechargeable sacral neuromodulation (SNM) devices for patients with urinary and bowel dysfunction. In 2018, Longitude Capital led a $40 million financing that preceded the company's IPO later that year. www.axonics.com.



    • Checkmate Pharmaceuticals is a clinical-stage biopharmaceutical company developing proprietary technology to harness the power of the immune system to combat cancer. In 2020, Longitude Capital and Novo Holdings led the company's $85 million Series C financing that preceded the company's IPO later in the year. www.checkmatepharma.com.



    • Inflazome is a biotechnology company developing small molecules that block harmful inflammation by targeting inflammasomes, protein complexes that generate signals in order to activate an immune response. In 2020, Inflazome announced its acquisition by Roche for an upfront payment of €380 million, and potential predetermined milestone payments. www.inflazome.com.



    • Inozyme Pharma is a rare disease pharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization. Longitude Capital led Inozyme's $49 million Series A financing in 2017, and was joined by New Enterprise Associates, Novo Holdings, and Sanofi Ventures. Inozyme completed its initial public offering in 2020. www.inozyme.com.



    • KaNDy Therapeutics was a UK-based clinical-stage company focused on optimizing the potential of its unique NK-1,3 receptor antagonist NT-814 to treat common, chronic debilitating conditions related to menopause. In 2020, KaNDy Therapeutics was acquired by Bayer Pharmaceuticals for an upfront payment of $425 million, and a potential $450 million in R&D, regulatory milestones and additional commercial milestones.



    • Molecular Templates is a clinical-stage company focused on the discovery and development of targeted biologic therapeutics. In 2017, Longitude Capital led Molecular Template's $40 million PIPE (private investment into public equity) financing. Molecular Templates now has strategic collaborations with Takeda Pharmaceuticals and Vertex Pharmaceuticals. www.mtem.com.



    • Poseida Therapeutics is a clinical-stage biopharmaceutical company dedicated to utilizing its proprietary gene engineering platform technologies to create next generation cell and gene therapeutics with the capacity to cure various cancers. Longitude Capital led Poseida's $30 million Series B financing in 2018. Poseida completed its IPO in 2020. www.poseida.com.



    • Vaxcyte is a next-generation vaccine company seeking to improve global health by developing superior and novel vaccines designed to prevent or treat some of the most common and deadly infectious diseases worldwide. Longitude Capital invested in Vaxcyte's $22 million Series A financing and served on the Board of the company through its IPO in 2020. www.vaxcyte.com.

    About Longitude Capital Recent Investments

    • Eargo is a medical device company dedicated to improving the quality of life of people with hearing loss. Longitude Capital and Gilde Healthcare led Eargo's $81 million Series E financing in 2020. www.eargo.com.



    • Epirium Bio is a clinical-stage biopharmaceutical company that uses insights related to the biology of mitochondrial function and tissue regeneration to pursue novel and clinically significant therapeutic approaches for neuromuscular, neurodegenerative, and mitochondrial disorders. Longitude Capital and ARCH Venture Partners led Epirium's $85 million Series A financing in 2019. www.epirium.com.



    • Polares Medical is a clinical-stage medical technology company focused on the development of a unique trans-catheter mitral valve hemi-replacement system to treat patients suffering from mitral regurgitation (MR). Longitude Capital led Polares Medical's $40 million Series B financing in 2020. www.polaresmedical.com.



    • WelbeHealth is a services company dedicated to unlocking the full potential of vulnerable seniors through PACE (Program of All-Inclusive Care for the Elderly), a comprehensive medical and social care model. Longitude Capital and .406 Ventures led WelbeHealth's $30 million Series C in 2020. www.welbehealth.com.

    Source: Longitude Capital

    Contact Information
    
    Maggie Jamison
    Longitude Capital
    650-854-5700 
    mjamison@longitudecapital.com

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  23. Upsized IPO in July 2020 raised $128.8 million in gross proceeds

    Submitted IND for INZ-701 for the treatment of ENPP1 deficiency; currently on FDA clinical hold pending completion of ongoing GLP toxicology studies

    Initiation of INZ-701 Phase 1/2 clinical trials anticipated in early 2021, as previously planned

    BOSTON, Sept. 03, 2020 (GLOBE NEWSWIRE) --  Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton, today reported financial results for the second quarter ended June 30, 2020 and provided recent business highlights.

    "We have made substantial progress over the past quarter…

    Upsized IPO in July 2020 raised $128.8 million in gross proceeds

    Submitted IND for INZ-701 for the treatment of ENPP1 deficiency; currently on FDA clinical hold pending completion of ongoing GLP toxicology studies

    Initiation of INZ-701 Phase 1/2 clinical trials anticipated in early 2021, as previously planned

    BOSTON, Sept. 03, 2020 (GLOBE NEWSWIRE) --  Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton, today reported financial results for the second quarter ended June 30, 2020 and provided recent business highlights.

    "We have made substantial progress over the past quarter and in July, including completing our upsized initial public offering in July, acquiring additional ENPP1 deficiency program assets, and submitting our first investigational new drug application (IND) for INZ-701 for the treatment of ENPP1 deficiency," said Axel Bolte, MSc, MBA, co-founder, president and chief executive officer of Inozyme Pharma. "FDA has requested final study reports for our ongoing three-month GLP toxicology studies in mice and non-human primates and has placed our planned Phase 1/2 clinical trial of INZ-701 in adult patients with ENPP1 deficiency in the United States on clinical hold pending submission of the reports. We expect to be able to submit these reports in the fourth quarter of this year and initiate our clinical program, as we had planned, in early 2021."

    Recent Business Highlights

    • Completed upsized Initial Public Offering – In July 2020, Inozyme completed its initial public offering of 8,050,000 shares of common stock at a public offering price of $16.00 per share, including the full exercise of the underwriter's option to purchase additional shares. Gross proceeds from the IPO were $128.8 million and net proceeds from the offering, after deducting underwriting discounts, commissions and offering expenses, were approximately $116.5 million.

       
    • Submitted an IND for INZ-701 - On July 30, 2020, Inozyme submitted its first IND for INZ-701 for the treatment of ENPP1 deficiency to the U.S. Food and Drug Administration (FDA). At the end of the 30-day FDA review period, the Company was notified that the IND was placed on clinical hold, pending submission of the final study report for its ongoing three-month toxicology studies in mice and non-human primates (NHPs) being performed in accordance with Good Laboratory Practices (GLP) regulations. Inozyme initiated these GLP toxicology studies prior to submission of the IND and expects to complete the studies and have reports available in the fourth quarter of 2020. The FDA did not request any additional preclinical data to resolve the clinical hold other than the submission of the final study report for the three-month GLP toxicology studies in mice and NHPs. Subject to the submission of the final study reports for the three-month toxicology studies as recommended by the FDA and the successful resolution of the clinical hold, the Company expects to initiate its Phase 1/2 trials in early 2021 and report initial safety and biomarker data in 2021, as it had planned.



    • Expanded and strengthened Board of Directors with appointment of Doug Treco, Ph.D., as Chairman and Lynne Sullivan, MST as an Independent Director – In May 2020, Doug Treco joined Inozyme's Board as chairman and Lynne Sullivan joined the Board as an independent director. Dr. Treco co-founded Ra Pharmaceuticals, Inc., where he was chief executive officer and a member of the Board of Directors from its inception until the company was acquired in April 2020.



      Ms. Sullivan is currently the chief financial officer for UNITY Biotechnology, and she previously served as the chief financial officer of Compass Therapeutics and as a senior vice president of finance at Biogen, where she spent 11 years, with global responsibility for Corporate Finance, Financial Planning and Analysis and Corporate Tax.
    • Acquired ENPP1 program assets from Alexion Pharmaceuticals – In July 2020, Inozyme announced the acquisition of intellectual property and assets from Alexion Pharmaceuticals focusing on ENPP1 gene deficiencies. The acquisition complements Inozyme's ongoing development of INZ-701 and expands the Company's intellectual property portfolio.

       
    • Initiated disease burden study in ENPP1 deficiency and ABCC6 deficiency – In May 2020, Inozyme and GACI Global, a patient advocacy organization dedicated to bettering the lives of families affected by Generalized Arterial Calcification of Infancy and/or Autosomal Recessive Hypophosphatemic Rickets Type 2 (GACI/ARHR2), announced the initiation of a study to characterize the burden of disease and understand the systemic progression of disease for the rare genetic diseases of both ENPP1 deficiency and ABCC6 deficiency from the perspective of a patient and/or parent.

       
    • Expanded physical footprint with move to new office – To meet the demands of anticipated growth, Inozyme expanded and enhanced its physical office space in August 2020.

    Second Quarter 2020 Financial Results

    • Cash Position – Cash, cash equivalents and short-term investments were $63.9 million as of June 30, 2020, as compared to $40.8 million as of March 31, 2020. Total cash, cash equivalents and short-term investments on June 30, 2020 does not include total net proceeds of approximately $116.5 million from the Company's IPO in July 2020. Based on its current plans, the Company expects that its existing cash, cash equivalents and short-term investments, including the proceeds from its July 2020 IPO, will be sufficient to enable it to fund its operating expenses and capital expenditure requirements into the second half of 2022.

       
    • Research and Development (R&D) Expenses – R&D expenses were $7.9 million for the second quarter ended June 30, 2020, compared to $3.5 million for the same period in 2019. The increase was primarily due to higher consulting and professional fees related to the planned filing of an IND for INZ-701, preclinical studies and clinical preparation activities with the Company's CRO, and growth in the number of R&D employees.

       
    • General and Administrative (G&A) Expenses – G&A expenses were $1.7 million for the second quarter ended June 30, 2020, compared to $1.1 million for the same period in 2019. The increase was primarily due to the growth in the number of G&A employees, an increase in legal fees related to patents and new contracts, and generally higher fees in areas such as audit, tax and information technology to support the Company's growth.

       
    • Net Loss – Net loss was $9.5 million, or $7.57 loss per share, for the second quarter ended June 30, 2020, compared to $4.2 million, or $3.57 loss per share, for the same period in 2019.

    About Inozyme Pharma

    Inozyme Pharma is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of potentially first-in-class therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 deficiencies.

    Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation, and timing, of our future clinical trials and our research and development programs, the availability of preclinical study and clinical trial data and the period over which we believe that our existing cash and cash equivalents will be sufficient to fund our operating expenses. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to successfully resolve the clinical hold with regard to its planned Phase 1/2 clinical trial of INZ-701 for ENPP1 deficiency; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain and protect intellectual property rights related to its product candidates; manage expenses; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Condensed Consolidated Balance Sheet Data

     (Unaudited)

    (in thousands)

      June 30,

    2020
     December 31,

    2019
    Cash, cash equivalents and short-term investments $63,867  $47,132 
    Total assets  68,511   47,944 
    Total liabilities  6,965   3,236 
    Convertible preferred stock  111,565   77,927 
    Accumulated deficit  (51,863)  (34,652)
    Total stockholders' deficit  (50,019)  (33,219)
         

    Condensed Consolidated Statements of Operations and Comprehensive Loss

    (Unaudited)

    (in thousands, except share and per share data)

      Three Months Ended June 30, Six Months Ended June 30,
       2020   2019   2020   2019 
    Operating expenses:        
    Research and development $7,877  $3,489  $14,283  $7,623 
    General and administrative  1,671   1,064   3,171   2,094 
    Total operating expenses  9,548   4,553   17,454   9,717 
    Loss from operations  (9,548)  (4,553)  (17,454)  (9,717)
    Other income (expense):        
    Interest income  71   394   242   604 
    Other income (expense), net  4   (14)  1   (31)
    Other income (expense), net  75   380   243   573 
    Net loss $(9,473) $(4,173) $(17,211) $(9,144)
    Other comprehensive (loss) income:        
    Unrealized (losses) gains on available-for-sale securities  (15)  10   8   12 
    Total other comprehensive (loss) income  (15)  10   8   12 
    Comprehensive loss $(9,488) $(4,163) $(17,203) $(9,132)
    Net loss attributable to common stockholders—basic

       and diluted
     $(9,473) $(4,173) $(17,211) $(9,144)
    Net loss per share attributable to common

       stockholders—basic and diluted
     $(7.57) $(3.57) $(14.01) $(7.83)
    Weighted-average common shares outstanding—basic

       and diluted
      1,251,244   1,170,480   1,228,296   1,167,346 
             

    Contacts

    Investors:

    Solebury Trout

    Mike Biega

    (617) 221 9660

    mbiega@soleburytrout.com

    Media:

    SmithSolve

    Alex Van Rees

    (973) 442-1555 ext. 111

    alex.vanrees@smithsolve.com

    Primary Logo

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  24. BOSTON, July 30, 2020 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. ("Inozyme") (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization, today announced the closing of its initial public offering of 7,000,000 shares of common stock at a public offering price of $16.00 per share on July 28, 2020 and the closing of the sale of 1,050,000 additional shares of common stock on July 30, 2020 following the exercise in full by the underwriters of their option to purchase additional shares. Aggregate gross proceeds from the initial public offering, including the option shares, totaled $128.8 million, before underwriting discounts and commissions and offering expenses payable…

    BOSTON, July 30, 2020 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. ("Inozyme") (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization, today announced the closing of its initial public offering of 7,000,000 shares of common stock at a public offering price of $16.00 per share on July 28, 2020 and the closing of the sale of 1,050,000 additional shares of common stock on July 30, 2020 following the exercise in full by the underwriters of their option to purchase additional shares. Aggregate gross proceeds from the initial public offering, including the option shares, totaled $128.8 million, before underwriting discounts and commissions and offering expenses payable by Inozyme. All shares were offered and sold by Inozyme.

    Inozyme's common stock began trading on the Nasdaq Global Select Market under the ticker symbol "INZY" on July 24, 2020.

    BofA Securities, Cowen and Piper Sandler acted as joint book-running managers for the offering. Wedbush PacGrow acted as lead manager for the offering.

    A registration statement relating to the offering of these securities was declared effective by the Securities and Exchange Commission (the "SEC") on July 23, 2020. Copies of the registration statement can be accessed by visiting the SEC website at www.sec.gov. This offering was made only by means of a prospectus. A copy of the final prospectus relating to the offering may be obtained from BofA Securities, NC1-004-03-43, 200 North College Street, 3rd floor, Charlotte, NC 28255-0001, Attn: Prospectus Department, or by email at dg.prospectus_requests@bofa.com; Cowen and Company, LLC, c/o Broadridge Financial Solutions, Attn: Prospectus Department, 1155 Long Island Avenue, Edgewood, NY 11717, by email at PostSaleManualRequests@broadridge.com or by telephone at (833) 297-2926; or Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, via telephone at (800) 747-3924 or via email at prospectus@psc.com.

    This press release does not constitute an offer to sell, or the solicitation of an offer to buy, securities, nor shall there be any sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of that jurisdiction.

    About Inozyme Pharma

    Inozyme Pharma is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 deficiencies.

    Contacts

    Investors:

    Inozyme Pharma

    Axel Bolte, Co-founder, President and CEO

    (857) 330-4345

    axel.bolte@inozyme.com

    Media:

    SmithSolve

    Alex Van Rees

    (973) 442-1555 ext. 111

    alex.vanrees@smithsolve.com

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